Trial Outcomes & Findings for Efficacy and Safety of PD-0332991 in Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib (NCT NCT01907607)
NCT ID: NCT01907607
Last Updated: 2025-08-24
Results Overview
Efficacy is assessed based on 4-month non progression. Non progression is defined as complete or partial response (CR, PR) or stable disease (SD), using the Response Evaluation Criteria in Solid Tumors (RECIST v1.1). Non-progression rate will be calculated as the number of alive and progression free patients divided by the number of eligible and assessable patients for the efficacy analysis. Eligible and assessable populations are described in corresponding section of protocol. As recommended by RECIST v1.1, all claimed response will be centrally reviewed by an expert of the study. The results of the centralized radiological review will be used for the analysis of the primary endpoint.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
29 participants
Primary outcome timeframe
16 weeks after first administration of treatment
Results posted on
2025-08-24
Participant Flow
Participant milestones
| Measure |
PD-0332991
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Overall Study
STARTED
|
29
|
|
Overall Study
COMPLETED
|
29
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
PD-0332991
n=29 Participants
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Age, Continuous
|
66 years
n=29 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=29 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=29 Participants
|
|
Region of Enrollment
France
|
29 participants
n=29 Participants
|
PRIMARY outcome
Timeframe: 16 weeks after first administration of treatmentPopulation: Population evaluable for efficacy: All patients eligible and for whom the following conditions are satisfied: (i) Received at least one complete or two incomplete treatment cycles, (ii) At least one disease measurement recorded not less than four weeks after treatment onset.
Efficacy is assessed based on 4-month non progression. Non progression is defined as complete or partial response (CR, PR) or stable disease (SD), using the Response Evaluation Criteria in Solid Tumors (RECIST v1.1). Non-progression rate will be calculated as the number of alive and progression free patients divided by the number of eligible and assessable patients for the efficacy analysis. Eligible and assessable populations are described in corresponding section of protocol. As recommended by RECIST v1.1, all claimed response will be centrally reviewed by an expert of the study. The results of the centralized radiological review will be used for the analysis of the primary endpoint.
Outcome measures
| Measure |
PD-0332991
n=23 Participants
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Number of Participants With Non Progression at 4 Months
|
3 Participants
|
SECONDARY outcome
Timeframe: 16 weeks after first administration of treatmentPopulation: Population evaluable for efficacy: All patients eligible and for whom the following conditions are satisfied: (i) Received at least one complete or two incomplete treatment cycles, (ii) At least one disease measurement recorded not less than four weeks after treatment onset.
Objective response is defined as complete response (CR) or partial response (PR) according to RECIST v1.1 Efficacy is assessed based on objective response at 4-month. Objective response is defined as complete or partial response (CR, PR) using the Response Evaluation Criteria in Solid Tumors (RECIST v1.1). Objective reponse will be calculated as the number of alive patients with objective reponse divided by the number of eligible and assessable patients for the efficacy analysis. Eligible and assessable populations are described in corresponding section of protocol. As recommended by RECIST v1.1, all claimed response will be centrally reviewed by an expert of the study. The results of the centralized radiological review will be used for the analysis.
Outcome measures
| Measure |
PD-0332991
n=23 Participants
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Number of Participants With Objective Response at 4 Months
|
0 Participants
|
SECONDARY outcome
Timeframe: up to 18 months following first administration of treatmentPopulation: Population evaluable for efficacy: All patients eligible and for whom the following conditions are satisfied: (i) Received at least one complete or two incomplete treatment cycles, (ii) At least one disease measurement recorded not less than four weeks after treatment onset.
Progression-free survival time is defined as the time from the first administration of treatment to progression (as per RECIST v1.1) or death of any cause, whichever occurs first. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Outcome measures
| Measure |
PD-0332991
n=23 Participants
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Efficacy Assessment of PD-0332991 in Terms of Progression-free Survival Time
|
1.74 Months
Interval 0.92 to 3.45
|
SECONDARY outcome
Timeframe: up to 24 months following first administration of treatmentPopulation: Population evaluable for efficacy: All patients eligible and for whom the following conditions are satisfied: (i) Received at least one complete or two incomplete treatment cycles, (ii) At least one disease measurement recorded not less than four weeks after treatment onset.
Overall survival is defined as the time from the first administration of treatment to death.
Outcome measures
| Measure |
PD-0332991
n=23 Participants
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Efficacy Assessment of PD-0332991 in Terms of Overall Survival Time
|
19.52 months
Interval 10.94 to 22.8
|
Adverse Events
PD-0332991
Serious events: 12 serious events
Other events: 29 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
PD-0332991
n=29 participants at risk
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
General disorders
Flu-like syndrom
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Left ilio femoral phebilits
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Blood and lymphatic system disorders
Anemia
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Investigations
Neutropenia
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Renal insuficiency
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Pulmonary embolism
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Hemoperitoneum
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Phlebitis
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Infections and infestations
Urinary infection
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Deterioration of renal function
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Bladder globe
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Gastric hemorrhage
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Gastric pain
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Disease progression
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Renal insufficiency
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
|
General disorders
Sudden death at home
|
3.4%
1/29 • Number of events 1 • up to 24 months following first administration of treatment
|
Other adverse events
| Measure |
PD-0332991
n=29 participants at risk
Adult patients with Advanced Gastrointestinal Stromal Tumors Refractory to Imatinib and Sunitinib.
PD-0332991 is formulated as gelatin capsules of 100 mg and 25 mg respectively.
PD-0332991 will be administrated orally, formulated as gelatin capsules of 100 mg and 25 mg respectively.: PD-0332991 dosed on a flat scale of 125 mg (1 capsule x 100 mg/day, 1 capsule x25 mg/day) will be administrated orally o.d on a 21 days on / 7 days off dosing schedule. One cycle is considered to consist of 4 weeks of PD-0332991 administration.
Patients should be instructed to administrate PD-0332991 with a sufficient amount of water at least 1 hour prior to a meal or at least 2 hours following a meal and to swallow the required number of capsules at approximately the same time on each day.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
58.6%
17/29 • Number of events 22 • up to 24 months following first administration of treatment
|
|
Cardiac disorders
Sinus tachycardia
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Eye disorders
Eye disorders - Other, specify
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
17.2%
5/29 • Number of events 5 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Constipation
|
24.1%
7/29 • Number of events 7 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Diarrhea
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Dysphagia
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Mucositis oral
|
17.2%
5/29 • Number of events 6 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Nausea
|
20.7%
6/29 • Number of events 7 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Stomach pain
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Vomiting
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
General disorders
Edema limbs
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
General disorders
Fatigue
|
34.5%
10/29 • Number of events 11 • up to 24 months following first administration of treatment
|
|
General disorders
Fever
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
General disorders
Flu like symptoms
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
General disorders
Pain
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
General disorders
General disorders and administration site conditions
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Investigations
Creatinine increased
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Investigations
Neutrophil count decreased
|
41.4%
12/29 • Number of events 17 • up to 24 months following first administration of treatment
|
|
Investigations
Platelet count increased
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Investigations
White blood cell decreased
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Metabolism and nutrition disorders
Anorexia
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
10.3%
3/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor pain
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Psychiatric disorders
Insomnia
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Urinary retention
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Renal and urinary disorders
Renal and urinary disorders - Other
|
13.8%
4/29 • Number of events 4 • up to 24 months following first administration of treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Skin and subcutaneous tissue disorders
other
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Hypertension
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Phlebitis
|
6.9%
2/29 • Number of events 2 • up to 24 months following first administration of treatment
|
|
Vascular disorders
Thromboembolic event
|
10.3%
3/29 • Number of events 3 • up to 24 months following first administration of treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place