Trial Outcomes & Findings for Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis (NCT NCT01981551)

NCT ID: NCT01981551

Last Updated: 2024-02-06

Results Overview

Complete Response + Partial Response was determined by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

20 months

Results posted on

2024-02-06

Participant Flow

Participant milestones

Participant milestones
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Overall Study STARTED	17
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Overall Study Refused further treatment	8
Overall Study Adverse Event	1

Baseline Characteristics

Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 Participants PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=99 Participants
Age, Categorical >=65 years	1 Participants n=99 Participants
Age, Continuous	39.02 years STANDARD_DEVIATION 14.47 • n=99 Participants
Sex: Female, Male Female	14 Participants n=99 Participants
Sex: Female, Male Male	3 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	17 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	2 Participants n=99 Participants
Race (NIH/OMB) White	15 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	17 participants n=99 Participants
Mean MD Anderson Symptom Inventory Scores at Baseline Symptom Severity Score	2.0 Scores on a scale STANDARD_DEVIATION 1.39 • n=99 Participants
Mean MD Anderson Symptom Inventory Scores at Baseline Symptom Interference Score	2.94 Scores on a scale STANDARD_DEVIATION 2.50 • n=99 Participants

PRIMARY outcome

Timeframe: 20 months

Outcome measures

Outcome measures
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 Participants PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Number of Participants With a Complete Response (CR) + Partial Response (PR) Complete Response	0 Participants
Number of Participants With a Complete Response (CR) + Partial Response (PR) Partial Response	5 Participants
Number of Participants With a Complete Response (CR) + Partial Response (PR) Not Evaluable	1 Participants
Number of Participants With a Complete Response (CR) + Partial Response (PR) Stable Disease	11 Participants
Number of Participants With a Complete Response (CR) + Partial Response (PR) Progressive Disease	0 Participants

SECONDARY outcome

Timeframe: Date treatment consent signed to date off study, approximately 66 months and 27 days.

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 Participants PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Number of Participants With Serious and Non-serious Adverse Events	17 Participants

SECONDARY outcome

Timeframe: Baseline

Tumor and blood samples were obtained from participants and genotyped for somatic and germline mutations.

Outcome measures

Outcome measures
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 Participants PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes	15 Participants

SECONDARY outcome

Timeframe: At baseline prior to drug administration and at least every 6 cycles of drug (18 weeks) up to 20 months of treatment.

The MD Anderson Symptoms Inventory (MDSAI) questionnaire includes questions regarding 13 symptoms commonly experienced by patients with cancer and 6 additional items that assess the extent to which these symptoms interfered with how patients felt and were able to function. The 0-10 scale (0=none, 1-4=mild, 5-6=moderate, and 7-10=severe) assesses how patients felt and were able to function over the previous 24 hours. A component score representing symptom severity is obtained by taking the average of the 13 symptom items (e.g., pain, fatigue, etc.) together. A component score representing symptom distress is obtaining by averaging the 6 symptom interference items (e.g., general activity, mood, etc.).

Outcome measures

Outcome measures
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 Participants PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Mean MD Anderson Symptom Inventory Scores After Treatment Symptom Severity Score	1.37 Scores on a scale Standard Deviation 1.46
Mean MD Anderson Symptom Inventory Scores After Treatment Symptom Interference Score	1.57 Scores on a scale Standard Deviation 2.69

Adverse Events

PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis

Serious events: 9 serious events

Other events: 17 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 participants at risk PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Immune system disorders Allergic reaction	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Back pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Blood bilirubin increased	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Bronchospasm	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Constipation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Fever	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Vascular disorders Hematoma	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Renal and urinary disorders Hematuria	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Injury, poisoning and procedural complications - Other, specify	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, squamous cell carcinoma	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Stroke	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Surgical and medical procedures Surgical and medical procedures - Other, hysterectomy	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Vaginal inflammation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.

Other adverse events

Other adverse events
Measure	PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis n=17 participants at risk PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles PF-03084014: Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Gastrointestinal disorders Abdominal pain	47.1% 8/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Activated partial thromboplastin time prolonged	23.5% 4/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Alanine aminotransferase increased	52.9% 9/17 • Number of events 35 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Alkaline phosphatase increased	29.4% 5/17 • Number of events 16 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Immune system disorders Allergic rhinitis	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Alopecia	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Amnesia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Blood and lymphatic system disorders Anemia	52.9% 9/17 • Number of events 23 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Anorexia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Anxiety	17.6% 3/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Arthralgia	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Arthritis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Aspartate aminotransferase increased	70.6% 12/17 • Number of events 32 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Atrial fibrillation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Back pain	29.4% 5/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Bloating	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Blood bilirubin increased	17.6% 3/17 • Number of events 18 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Blood gonadotrophin abnormal	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Blurred vision	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Bronchial infection	5.9% 1/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Bruising	29.4% 5/17 • Number of events 7 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Bullous dermatitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Cardiac disorders - Other, potential murmur/"whooshing"	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Chest wall pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Chills	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Cholesterol high	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Cognitive disturbance	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Concentration impairment	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Confusion	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Conjunctivitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Constipation	29.4% 5/17 • Number of events 7 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Cough	35.3% 6/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Creatinine increased	17.6% 3/17 • Number of events 13 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Renal and urinary disorders Cystitis noninfective	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Dehydration	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Depression	17.6% 3/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Diarrhea	88.2% 15/17 • Number of events 39 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Dizziness	23.5% 4/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Dry eye	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Dry mouth	41.2% 7/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Dry skin	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Dysgeusia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Dysmenorrhea	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Dyspepsia	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Dysphagia	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Dyspnea	11.8% 2/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Ear and labyrinth disorders - Other, eustachian tube dysfunction	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Ear pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Edema limbs	35.3% 6/17 • Number of events 15 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Edema trunk	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Electrocardiogram QT corrected interval prolonged	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Endocrine disorders Endocrine disorders - Other, elevated glucose level	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Enterocolitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Epistaxis	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Erythema multiforme	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, eye injury	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, left eye swelling & discharge	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, macular degeneration - left eye	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, vision changes	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, visual disturbance	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Eye disorders - Other, exopthalmos bilateral	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Facial pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Fatigue	52.9% 9/17 • Number of events 14 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Fever	35.3% 6/17 • Number of events 13 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Flatulence	11.8% 2/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Eye disorders Floaters	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Flu like symptoms	35.3% 6/17 • Number of events 7 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Fracture	23.5% 4/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gastroesophageal reflux disease	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gastrointestinal disorders - Other, abdominal cramping	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gastrointestinal disorders - Other, abdominal discomfort	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gastrointestinal disorders - Other, canker sores	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gastrointestinal disorders - Other, gastroenteritis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Gingival pain	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Headache	76.5% 13/17 • Number of events 22 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Vascular disorders Hematoma	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Hemoglobin increased	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Hemorrhoids	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Hip fracture	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Hirsutism	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Hoarseness	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Vascular disorders Hot flashes	47.1% 8/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypercalcemia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hyperglycemia	17.6% 3/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Hyperhidrosis	23.5% 4/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hyperkalemia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypermagnesemia	11.8% 2/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypernatremia	11.8% 2/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Vascular disorders Hypertension	41.2% 7/17 • Number of events 28 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypoalbuminemia	29.4% 5/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypocalcemia	52.9% 9/17 • Number of events 21 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypoglycemia	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypokalemia	52.9% 9/17 • Number of events 14 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypomagnesemia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hyponatremia	47.1% 8/17 • Number of events 17 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Hypophosphatemia	76.5% 13/17 • Number of events 46 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, fungal infection (ear)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, oral infections	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, rhinitis infective	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, sinus infection	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, upper respiratory infection	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Infections and infestations - Other, viral gastroenteritis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Infusion related reaction	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Infusion site extravasation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Injection site reaction	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Injury, poisoning and procedural complications - Other, injury, abdomen contusion	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Injury, poisoning and procedural complications - Other, injury, swollen mouth	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Insomnia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Investigations - Other, hypophosphatemia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Irregular menstruation	23.5% 4/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Joint effusion	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Laryngitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Libido decreased	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Lip infection	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Lung infection	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Lymphocyte count decreased	70.6% 12/17 • Number of events 34 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Lymphocyte count increased	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Malaise	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Memory impairment	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Menorrhagia	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Metabolism and nutrition disorders Metabolism and nutrition disorders - Other, hyperphosphatemia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Middle ear inflammation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Mucositis oral	35.3% 6/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, bone spur	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, right deltoid muscle tenderness	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, stress injury	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, tendinitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, tendinitis left groin/thigh	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Myalgia	35.3% 6/17 • Number of events 10 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Nail infection	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Nasal congestion	23.5% 4/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Nausea	76.5% 13/17 • Number of events 27 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, cyst	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, cyst - right arm	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, popliteal cysts	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, R paraspinal form tumor	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, rt groin area	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Nervous system disorders - Other, lightheadedness	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Neutrophil count decreased	17.6% 3/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Non-cardiac chest pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Oral hemorrhage	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Oral pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Osteoporosis	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
General disorders Pain	47.1% 8/17 • Number of events 18 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Pain in extremity	29.4% 5/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Pain of skin	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Palpitations	11.8% 2/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Paresthesia	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Pelvic pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Periodontal disease	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Peripheral sensory neuropathy	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Pharyngitis	11.8% 2/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Platelet count decreased	23.5% 4/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Pleural effusion	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Hepatobiliary disorders Portal vein thrombosis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Postnasal drip	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Injury, poisoning and procedural complications Postoperative hemorrhage	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Productive cough	11.8% 2/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Pruritus	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Psychiatric disorders - Other, Mood swings	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Rash acneiform	41.2% 7/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Rash maculo-papular	58.8% 10/17 • Number of events 19 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Renal and urinary disorders Renal and urinary disorders - Other, elevated BUN	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Renal and urinary disorders Renal and urinary disorders - Other, kidney stones	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, amenorrhea	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, breast tenderness	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, elevated FSH	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, elevated LH	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, genital infection	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, menstrual spotting	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, menstruation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Reproductive system and breast disorders - Other, R breat lump	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other, cold	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other, dry nasal passages	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other, pneumonia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other, sinus congestion	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Nervous system disorders Seizure	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Sinus tachycardia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Sinusitis	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, cyst	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, folliculitis	23.5% 4/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, skin keratosis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, skin lesions	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, small hard lump/Rt upper thigh	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, left groin cystic lesion	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, subcutaneous cysts	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Skin infection	41.2% 7/17 • Number of events 17 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Skin ulceration	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Musculoskeletal and connective tissue disorders Soft tissue necrosis upper limb	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Sore throat	23.5% 4/17 • Number of events 7 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Stomach pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Cardiac disorders Supraventricular tachycardia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Surgical and medical procedures Surgical and medical procedures - Other, esophageal dilation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Surgical and medical procedures Surgical and medical procedures - Other, splenectomy	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Vascular disorders Thromboembolic event	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Tinnitus	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Tooth infection	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	29.4% 5/17 • Number of events 7 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Renal and urinary disorders Urinary frequency	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Urinary tract infection	11.8% 2/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Skin and subcutaneous tissue disorders Urticaria	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Vaginal dryness	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Vaginal infection	11.8% 2/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Vaginal inflammation	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Reproductive system and breast disorders Vaginal pain	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Vertigo	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Gastrointestinal disorders Vomiting	35.3% 6/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Ear and labyrinth disorders Watering eyes	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Weight gain	11.8% 2/17 • Number of events 14 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations Weight loss	23.5% 4/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Respiratory, thoracic and mediastinal disorders Wheezing	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Investigations White blood cell decreased	41.2% 7/17 • Number of events 17 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Infections and infestations Wound infection	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.
Psychiatric disorders Psychiatric disorders - Other, Dreams	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 66 months and 27 days.

Additional Information

Dr. Geraldine H O'Sullivan Coyne

National Cancer Institute

Phone: 240-781-3371

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place