Trial Outcomes & Findings for SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors (NCT NCT03433183)
NCT ID: NCT03433183
Last Updated: 2026-05-27
Results Overview
An evaluable patient will be classified as a responder if the patient achieves a partial response (PR), complete response (CR) or stable disease (SD) ≥ 4 cycles.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Up to 6 months
Results posted on
2026-05-27
Participant Flow
Participant milestones
| Measure |
Selumetinib and Sirolimus
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Overall Study
STARTED
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21
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Overall Study
COMPLETED
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21
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
Baseline characteristics by cohort
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Age, Categorical
<=18 years
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1 Participants
n=51 Participants
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Age, Categorical
Between 18 and 65 years
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18 Participants
n=51 Participants
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Age, Categorical
>=65 years
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2 Participants
n=51 Participants
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Age, Continuous
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41 years
n=51 Participants
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Sex: Female, Male
Female
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7 Participants
n=51 Participants
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Sex: Female, Male
Male
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14 Participants
n=51 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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1 Participants
n=51 Participants
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Race (NIH/OMB)
Asian
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1 Participants
n=51 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=51 Participants
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Race (NIH/OMB)
Black or African American
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3 Participants
n=51 Participants
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Race (NIH/OMB)
White
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15 Participants
n=51 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=51 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=51 Participants
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Region of Enrollment
United States
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21 participants
n=51 Participants
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PRIMARY outcome
Timeframe: Up to 6 monthsPopulation: Clinical Benefit Outcome info: * Estimation Parameter: Other (Proportion) * Estimated Value: 9.5% (or 0.095 if it wants a proportion) * Confidence Interval (2-sided): 95% CI: 1.67%, 32%
An evaluable patient will be classified as a responder if the patient achieves a partial response (PR), complete response (CR) or stable disease (SD) ≥ 4 cycles.
Outcome measures
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Clinical Benefit Rate of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
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0.095 Proportion
Interval 0.0167 to 0.32
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SECONDARY outcome
Timeframe: PFS is the duration of time from the start of treatment to the time of objective progression or death whichever happens first up to 4 years. OS is the duration of time from the start of treatment to the time of death; assessed up to 4 years.Population: PFS Outcome data: * Measure type: median time to event * Unit of measure: months * Estimated Value: 1.97 months * Confidence Interval: 95% CI (1.71, 3.65 months)
Determined using the Kaplan-Meier method with PFS at important time points reported along with 95% two sided confidence intervals.
Outcome measures
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Progression Free (PFS) and Overall Survival (OS)
PFS
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1.97 Months
Interval 1.71 to 3.65
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Progression Free (PFS) and Overall Survival (OS)
OS
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6.12 Months
Interval 3.55 to 10.39
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SECONDARY outcome
Timeframe: Up to 6 monthsTreatment-emergent adverse events occurring after initiation of selumetinib in combination with sirolimus were assessed and graded according to CTCAE v5.0. The safety population included all participants who received at least one dose of study treatment.
Outcome measures
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Anemia · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Anemia · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Infection, cecal · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Infection, cecal · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Infection, cecal · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Anemia · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Dyspnea · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Dyspnea · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Dyspnea · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypertension · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypertension · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypertension · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypokalemia · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypokalemia · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypokalemia · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypoxia · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypoxia · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypoxia · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypophosphatemia · Grade 3
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypophosphatemia · Grade 4
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Hypophosphatemia · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Mucositis, oral · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Mucositis, oral · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Mucositis, oral · No Grade 3-4 AE reported
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20 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Pleural effusion · Grade 3
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1 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Pleural effusion · Grade 4
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0 Participants
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Define and Describe the Toxicities of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST.
Pleural effusion · No Grade 3-4 AE reported
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20 Participants
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SECONDARY outcome
Timeframe: Up to 6 monthsPopulation: Change in Pain Interference from baseline to pre-cycle 2 (calculated as the score pre-cycle 2 minus the score at baseline). Positive values indicate increasing pain. Higher scores indicate worse pain The statistical test assesses if there is a significant change in pain interference from baseline to pre-cycle 2 Wilcoxon signed-rank test
Change in Pain Interference (PROMIS) from baseline to pre-cycle 2 (calculated as the score pre-cycle 2 minus the score at baseline). Positive values indicate increasing pain. Higher scores indicate worse pain The statistical test assesses if there is a significant change in pain interference from baseline to pre-cycle 2 Wilcoxon signed-rank test T-scores are standardized to the general population with a mean of 50 and standard deviation of 10. Higher scores indicate worse anxiety symptoms. A T-score of 55-60 indicates mild pain interference, 60-70 indicates moderate pain interference, and 70-80 indicates severe pain interference.
Outcome measures
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Assess the Impact on Pain Interference
Median at baseline
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61.5 PROMIS T-Score
Interval 56.2 to 64.7
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Assess the Impact on Pain Interference
Median pre Cycle 2
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55.9 PROMIS T-Score
Interval 52.5 to 61.3
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SECONDARY outcome
Timeframe: Up to 6 monthsChange in Pain Intensity, as assessed on the numerical rating scale 11, from baseline to pre-cycle 2 (calculated as the score pre-cycle 2 minus the score at baseline). Positive values indicate increasing pain. Higher scores indicate worse pain The statistical test assesses if there is a significant change in pain intensity from baseline to pre-cycle 2 Wilcoxon signed-rank test The Numerical Rating Scale-11 (NRS-11) is a self-report segmented 11-point numeric scale that assesses pain severity. It consists of a horizontal line with 0 representing "no pain" at the right end of the line and 10 representing "worst pain you can imagine" at the left end.
Outcome measures
| Measure |
Selumetinib and Sirolimus
n=21 Participants
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Assess the Impact on Pain Severity
Median at baseline
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6.0 Numerical Rating Scale-11 (NRS-11) score
Interval 2.5 to 9.5
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Assess the Impact on Pain Severity
Median pre Cycle 2
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5.0 Numerical Rating Scale-11 (NRS-11) score
Interval 2.5 to 6.5
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Adverse Events
Selumetinib and Sirolimus
Serious events: 21 serious events
Other events: 21 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Selumetinib and Sirolimus
n=21 participants at risk
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
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Gastrointestinal disorders
ABDOMINAL PAIN
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9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Infections and infestations
ADMITTED FOR WOUND EROSION WITH POSSIBLE SUPERINFECTION
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Blood and lymphatic system disorders
ANEMIA
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14.3%
3/21 • Number of events 3 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Infections and infestations
CECAL INFECTION
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Respiratory, thoracic and mediastinal disorders
DYSPNEA
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Gastrointestinal disorders
ENTEROCOLITIS
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Cardiac disorders
HYPERTENSION
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9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Renal and urinary disorders
HYPERURICEMIA
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Metabolism and nutrition disorders
HYPOKALEMIA
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9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Metabolism and nutrition disorders
HYPOPHOSPHATEMIA
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Respiratory, thoracic and mediastinal disorders
HYPOXIA
|
9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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General disorders
MULTI-ORGAN FAILURE
|
9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Respiratory, thoracic and mediastinal disorders
PLEURAL EFFUSION
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Infections and infestations
SEPSIS
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4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Cardiac disorders
SINUS TACHYCARDIA
|
4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Cardiac disorders
SUPRAVENTRICULAR TACHYCARDIA
|
4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
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Blood and lymphatic system disorders
THROMBOEMBOLIC EVENT
|
4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Infections and infestations
UPPER RESPIRATORY INFECTION
|
4.8%
1/21 • Number of events 1 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
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Infections and infestations
URINARY TRACT INFECTION
|
9.5%
2/21 • Number of events 2 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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General disorders
ASCITES
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
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Ear and labyrinth disorders
HEARING IMPAIRED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
LEUKOCYTOSIS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
LYMPHOCYTE COUNT DECREASED
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
ANOREXIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
MUCOSITIS ORAL
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
Other adverse events
| Measure |
Selumetinib and Sirolimus
n=21 participants at risk
A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.
Selumetinib: Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
Sirolimus: Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.
|
|---|---|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Hepatobiliary disorders
ALANINE AMINOTRANSFERASE INCREASED
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Hepatobiliary disorders
ALKALINE PHOSPHATASE INCREASED
|
33.3%
7/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
ANEMIA
|
33.3%
7/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
ANOREXIA
|
19.0%
4/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Hepatobiliary disorders
ASPARTATE AMINOTRANSFERASE INCREASED
|
28.6%
6/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
BLEEDING FROM ANKLE TUMOR SITE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
CHOLESTEROL HIGH
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Gastrointestinal disorders
CONSTIPATION
|
28.6%
6/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
CPK INCREASED
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
CREATININE INCREASED
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Gastrointestinal disorders
DIARRHEA
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Eye disorders
DRY EYE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Skin and subcutaneous tissue disorders
DRY SKIN
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DYSGEUSIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
FALL
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
FATIGUE
|
33.3%
7/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
FEVER
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
GENERALIZED MUSCLE WEAKNESS
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPERGLYCEMIA
|
38.1%
8/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPERPHOSPHATEMIA
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Cardiac disorders
HYPERTENSION
|
19.0%
4/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPERTRIGLYCERIDEMIA
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPOALBUMINEMIA
|
42.9%
9/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPOCALCEMIA
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPOKALEMIA
|
19.0%
4/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPOPHOSPHATEMIA
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
LYMPHOCYTE COUNT DECREASED
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
MOUTH SORES
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
MUCOSITIS ORAL
|
28.6%
6/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
MYALGIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Gastrointestinal disorders
NAUSEA
|
57.1%
12/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
NEUTROPHIL COUNT DECREASED
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Infections and infestations
PARONYCHIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
PLATELET COUNT DECREASED
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Skin and subcutaneous tissue disorders
RASH ACNEIFORM
|
42.9%
9/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Skin and subcutaneous tissue disorders
RASH MACULO-PAPULAR
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Skin and subcutaneous tissue disorders
SKIN ULCERATION
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Cardiac disorders
SUPRAVENTRICULAR TACHYCARDIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Gastrointestinal disorders
VOMITING
|
33.3%
7/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
WEIGHT LOSS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
WHITE BLOOD CELL DECREASED
|
28.6%
6/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
ACTIVATED PARTIAL THROMBOPLASTIN TIME PROLONGED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
ALLERGIC RHINITIS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Psychiatric disorders
ANXIETY
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
ASTHMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
BLOATING
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Skin and subcutaneous tissue disorders
BULLOUS DERMATITIS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
CHILLS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
CONCENTRATION IMPAIRMENT
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
CONFUSION
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DEHYDRATION
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Psychiatric disorders
DEPRESSION
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DIZZINESS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DRIED BLOOD IN EAR
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DRY MOUTH
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
DYSESTHESIA
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNEA
|
19.0%
4/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
EDEMA FACE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
EDEMA LIMBS
|
33.3%
7/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
EPISTAXIS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
FECAL INCONTINENCE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
FLU LIKE SYMPTOMS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
FRACTURE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
GAIT DISTURBANCE
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Gastrointestinal disorders
GASTROESOPHAGEAL REFLUX DISEASE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
GENITAL EDEMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
HEADACHE
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Renal and urinary disorders
HEMATURIA
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
HYDROCEPHALUS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPERKALEMIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPERMAGNESEMIA
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPOGLYCEMIA
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
HYPONATREMIA
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Cardiac disorders
HYPOTENSION
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
HYPOXIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
INR INCREASED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
INSOMNIA
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
JOINT RANGE OF MOTION DECREASED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
LETHARGY
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
LOCALIZED EDEMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
LYMPHEDEMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
MUSCLE WEAKNESS LOWER LIMB
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
NON-CARDIAC CHEST PAIN
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Psychiatric disorders
OCD
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Eye disorders
OPTIC GLIOMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
ORAL PAIN
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
OSTEOPOROSIS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PAIN
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PAIN IN EXTREMITY
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PARESTHESIA
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PERIORBITAL EDEMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Nervous system disorders
PERIPHERAL MOTOR NEUROPATHY
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Nervous system disorders
PERIPHERAL SENSORY NEUROPATHY
|
14.3%
3/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PHOTOPHOBIA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
PRESENTED WITH 2 BUMPS US DONE PROGRESSION NOT SUSPECTED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Psychiatric disorders
PSYCHIATRIC DISORDERS - OTHER, SPECIFY
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Renal and urinary disorders
RENAL CALCULI
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
SEROMA
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Cardiac disorders
SINUS TACHYCARDIA
|
28.6%
6/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Blood and lymphatic system disorders
THROMBOEMBOLIC EVENT
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
TRISMUS
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
General disorders
TUMOR PAIN
|
23.8%
5/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Infections and infestations
UPPER RESPIRATORY INFECTION
|
9.5%
2/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Renal and urinary disorders
URINARY RETENTION
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
VITAMIN D DEFICIENCY
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Injury, poisoning and procedural complications
WOUND COMPLICATION
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
|
Metabolism and nutrition disorders
CREATININE KINASE INCREASED
|
4.8%
1/21 • All-cause mortality was monitored from enrollment until death or end of follow-up, up to 4 years. Serious and other adverse events were assessed from first study treatment through 30 days after the last treatment or until resolution or stabilization of the adverse event (whichever occurs last), up to 4 years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place