Trial Outcomes & Findings for Vismodegib in Treating Patients With Steroid-Refractory Chronic Graft-Versus-Host Disease (NCT NCT02337517)
NCT ID: NCT02337517
Last Updated: 2021-07-19
Results Overview
Defined as the count of participants at six months with an absence of non relapse mortality (NRM), no recurrent malignancy, steroid dose at 6 months =\< 0.2 mg/kg/day of prednisone dose equivalent (PDE), and no addition of new systemic treatment for chronic graft-versus-host disease (GVHD) at six months after start of treatment.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
At 6 months
Results posted on
2021-07-19
Participant Flow
Participant milestones
| Measure |
Supportive Care (Vismodegib)
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Vismodegib in Treating Patients With Steroid-Refractory Chronic Graft-Versus-Host Disease
Baseline characteristics by cohort
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=99 Participants
|
|
Age, Continuous
|
53 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 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
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
6 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
|
|
Baseline Chronic GVHD Grade
Mild GVHD
|
0 Participants
n=99 Participants
|
|
Baseline Chronic GVHD Grade
Moderate GVHD
|
0 Participants
n=99 Participants
|
|
Baseline Chronic GVHD Grade
Severe GVHD
|
6 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: At 6 monthsDefined as the count of participants at six months with an absence of non relapse mortality (NRM), no recurrent malignancy, steroid dose at 6 months =\< 0.2 mg/kg/day of prednisone dose equivalent (PDE), and no addition of new systemic treatment for chronic graft-versus-host disease (GVHD) at six months after start of treatment.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Failure Free Survival (FFS)
|
3 Participants
|
SECONDARY outcome
Timeframe: Up to 1 month post last date of treatment (max time of approximately one year)The count of participants who experience adverse events (AE) and serious adverse events (SAE) while on treatment or within one month after discontinuing treatment.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Incidence of Adverse Events
Participants with any AE
|
6 Participants
|
|
Incidence of Adverse Events
Participants with any SAE
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline to up to 12 months after initiation of protocol therapyPopulation: Only 3 participants had NIH GVHD assessment for response at 6 months. Only 1 participant had NIH GVHD assessment for response at 12 months.
Chronic graft-versus-host disease (GVHD) severity was assessed at baseline with the National Institutes of Health (NIH) global score of chronic GVHD, which was defined using the 2014 NIH consensus criteria for diagnosis and staging of chronic GVHD (see Baseline Characteristics). Following initiation of treatment, the response to therapy was assessed as complete response (CR) (complete resolution of GVHD in one or more organ system) , partial response (PR) (improvement of GVHD in one or more organ system), stable disease (SD) (no change in GVHD), or progressive disease (PD) (worsened GVHD in one or more organ system), based on 2014 NIH consensus criteria for response assessment. GVHD was assessed monthly, with defined study endpoints at 6 and 12 months. Additionally, best overall response on the study is being reported to include response for patients who did not remain on study long enough to be included in the 6 month assessment.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Chronic Graft-versus-host Disease Response
Best Overall Response on study · Partial Response (PR)
|
6 Participants
|
|
Chronic Graft-versus-host Disease Response
Best Overall Response on study · CR, SD, and PD
|
0 Participants
|
|
Chronic Graft-versus-host Disease Response
Response at 6 months · Partial Response (PR)
|
3 Participants
|
|
Chronic Graft-versus-host Disease Response
Response at 6 months · CR, SD, and PD
|
0 Participants
|
|
Chronic Graft-versus-host Disease Response
Response at 12 months · Partial Response (PR)
|
1 Participants
|
|
Chronic Graft-versus-host Disease Response
Response at 12 months · CR, SD, and PD
|
0 Participants
|
SECONDARY outcome
Timeframe: At 1 yearRelapse will be defined as malignancy relapse or as the initiation of any unintended intervention including an unplanned taper of immunosuppressant therapy to prevent malignancy progression due to any signs of recurrent, residual or new malignant disease after transplantation. Non relapse mortality is the number of participant deaths due to any cause other than relapse.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
One-year Non Relapse Mortality (NRM)
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to one yearRelapse will be defined as malignancy relapse or as the initiation of any unintended intervention including an unplanned taper of immunosuppressant therapy to prevent malignancy progression due to any signs of recurrent, residual or new malignant disease after transplantation. This measure is reported as the count of participants who experienced a relapse of their primary malignancy within one year of starting study therapy.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
One Year Relapse
|
1 Participants
|
SECONDARY outcome
Timeframe: At 1 yearDefined as the count of participants at one year with an absence of non relapse mortality (NRM), no recurrent malignancy, steroid dose at 6 months =\< 0.2 mg/kg/day of prednisone dose equivalent (PDE), and no addition of new systemic treatment for chronic graft-versus-host disease (GVHD) at one year after start of treatment.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
One-year Failure Free Survival (FFS)
|
1 Participants
|
SECONDARY outcome
Timeframe: At 1 yearA count of participants who are not deceased at one year after initiation of study therapy.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
One-year Overall Survival
|
5 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearThe study proposed to evaluate clinical characteristics that could be associated with FFS. Each patient was categorized as either having FFS greater than 6 months or less than 6 months. Factors evaluated were whether or not the patient had high intensity conditional with total-body irradiation (TBI) before bone marrow transplant, whether or not the patient had \> 3 involved sites with chronic graft versus host disease (GVHD), whether or not the patient had lower gastrointestinal (GI) involvement by GVHD, and whether or not the patient had a Severe NIH global score at initiation of protocol treatment.
Outcome measures
| Measure |
Supportive Care (Vismodegib)
n=6 Participants
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: TBI before transplanat · Had Factor and FFS > 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: TBI before transplanat · Did not have factor and FFS > 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: TBI before transplanat · Had factor and FFS < 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: TBI before transplanat · Did not have factor and FFS < 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Greater than 3 sites involved with GVHD · Had Factor and FFS > 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Greater than 3 sites involved with GVHD · Did not have factor and FFS > 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Greater than 3 sites involved with GVHD · Had factor and FFS < 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Greater than 3 sites involved with GVHD · Did not have factor and FFS < 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Lower GI involvement with GVHD · Had Factor and FFS > 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Lower GI involvement with GVHD · Did not have factor and FFS > 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Lower GI involvement with GVHD · Had factor and FFS < 6 months
|
2 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Lower GI involvement with GVHD · Did not have factor and FFS < 6 months
|
1 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Severe NIH Global GVHD Score · Had Factor and FFS > 6 months
|
3 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Severe NIH Global GVHD Score · Did not have factor and FFS > 6 months
|
0 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Severe NIH Global GVHD Score · Had factor and FFS < 6 months
|
3 Participants
|
|
Clinical Characteristics Associated With Failure Free Survival (FFS)
Factor: Severe NIH Global GVHD Score · Did not have factor and FFS < 6 months
|
0 Participants
|
Adverse Events
Supportive Care (Vismodegib)
Serious events: 4 serious events
Other events: 6 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Supportive Care (Vismodegib)
n=6 participants at risk
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Injury, poisoning and procedural complications
Fracture
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Vascular disorders
Hypotension
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Pneumatosis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Infections and infestations
Sepsis
|
16.7%
1/6 • Number of events 2 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Weight loss
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
Other adverse events
| Measure |
Supportive Care (Vismodegib)
n=6 participants at risk
Patients receive vismodegib PO daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Renal and urinary disorders
Acute kidney injury
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Endocrine disorders
Adrenal insufficiency
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Psychiatric disorders
Agitation
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Alkaline phosphatase increased
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
66.7%
4/6 • Number of events 7 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Blood and lymphatic system disorders
Anemia
|
16.7%
1/6 • Number of events 2 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Anorexia
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • Number of events 2 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Bloating
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Skin and subcutaneous tissue disorders
Bullous dermatitis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
BUN elevated
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Cardiac disorders
Chest pain - cardiac
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
colonic polyps
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Concentration impairment
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Psychiatric disorders
Confusion
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Eye disorders
conjunctival irritation lower eye
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Cardiac disorders
Coronary atherosclerosis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Creatinine increased
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
crown of tooth detached
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Dental caries
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Psychiatric disorders
Depression
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Diarrhea
|
83.3%
5/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Dry mouth
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Dysgeusia
|
66.7%
4/6 • Number of events 7 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Dysphagia
|
16.7%
1/6 • Number of events 2 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
16.7%
1/6 • Number of events 2 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Blood and lymphatic system disorders
elevated neutrophils
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Encephalopathy
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Infections and infestations
Epstein Barr Virus
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Injury, poisoning and procedural complications
Fall
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
General disorders
Fatigue
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
General disorders
Fever
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Flatulence
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
General disorders
Flu like symptoms
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Injury, poisoning and procedural complications
Fracture
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Gastritis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
gastrointestinal disorder
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Hypernatremia
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
33.3%
2/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Vascular disorders
Hypotension
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Metabolism and nutrition disorders
Increased Appetite
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Psychiatric disorders
Insomnia
|
33.3%
2/6 • Number of events 3 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Infections and infestations
leg ulceration
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Lymphocyte count decreased
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Movements involuntary
|
50.0%
3/6 • Number of events 6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Muscle cramps
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
33.3%
2/6 • Number of events 3 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Nausea
|
83.3%
5/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Neutrophil count decreased
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Infections and infestations
Rhinitis infective
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Infections and infestations
Skin infection
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Spasticity
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Gastrointestinal disorders
Stomach pain
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Syncope
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Nervous system disorders
Tremor
|
33.3%
2/6 • Number of events 3 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Vascular disorders
Vasculitis
|
16.7%
1/6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
|
Investigations
Weight loss
|
50.0%
3/6 • Number of events 6 • From the first dose of study treatment until 30 days after the last dose, up to one year.
The occurrence of adverse events (AEs) was sought by non-directive questioning of the patient at each visit or phone contact during the study. Adverse events may also have been detected when they were volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60