Trial Outcomes & Findings for Study of Platelet Derived Growth Factor Receptor (PDGFR) in Recurrent Malignant Gliomas (NCT NCT01140568)

Last Updated: 2020-05-05

Results Overview

Progression was defined by McDonald Criteria: A 25% increase in the sum of products of all measurable lesions over smallest sum observed (over baseline if no decrease) using the same techniques as baseline, OR clear worsening of any evaluable disease, OR appearance of any new lesion/site, OR clear clinical worsening OR failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

38 participants

Primary outcome timeframe

6 months

Results posted on

2020-05-05

Participant Flow

38 people were enrolled in the study and 34 were treated with study drug. When patients came off treatment they moved into survival follow up.

Participant milestones

Participant milestones
Measure	Nilotinib Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Overall Study STARTED	38
Overall Study Screen Fail	4
Overall Study Treated With Study Drug	34
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	36

Reasons for withdrawal

Reasons for withdrawal
Measure	Nilotinib Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Overall Study disease progression	31
Overall Study Withdrawal by Subject	1
Overall Study Screen Fail	4

Baseline Characteristics

Study of Platelet Derived Growth Factor Receptor (PDGFR) in Recurrent Malignant Gliomas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nilotinib n=38 Participants Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=99 Participants
Age, Categorical >=65 years	10 Participants n=99 Participants
Sex: Female, Male Female	8 Participants n=99 Participants
Sex: Female, Male Male	30 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	4 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	34 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

PRIMARY outcome

Timeframe: 6 months

Population: 38 patients were enrolled, but only 34 were treated

Outcome measures

Outcome measures
Measure	Nilotinib n=34 Participants Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Number of Patients Who Had 6-month Progression-free Survival.	3 Participants

SECONDARY outcome

Timeframe: 5 years

Population: 38 patients enrolled, but only 34 received study drug

How many patients who had decrease in tumor size or complete disappearance of tumor.

Outcome measures

Outcome measures
Measure	Nilotinib n=34 Participants Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Overall Response Rate	1 Participants

Adverse Events

Nilotinib

Serious events: 3 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Nilotinib n=34 participants at risk Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Musculoskeletal and connective tissue disorders Musculoskeletal tissue disorder-other: Muscle Spasm	2.9% 1/34 • 8 years used clinicaltrials.gov definitions
Musculoskeletal and connective tissue disorders Generalized Muscle Weakness	2.9% 1/34 • 8 years used clinicaltrials.gov definitions
Nervous system disorders Hydrocephalus	2.9% 1/34 • 8 years used clinicaltrials.gov definitions

Other adverse events

Other adverse events
Measure	Nilotinib n=34 participants at risk Patients will take nilotinib twice daily at the standard dose of 400mg taken by mouth twice a day until disease progression or development of unacceptable side effects. nilotinib: 400mg po (orally) BID (twice daily)
Investigations Alkaline Phosphatase Increased	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Investigations ALT Increase	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Blood and lymphatic system disorders Anemia	11.8% 4/34 • 8 years used clinicaltrials.gov definitions
Musculoskeletal and connective tissue disorders Arthralgia	11.8% 4/34 • 8 years used clinicaltrials.gov definitions
Investigations AST Increased	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Psychiatric disorders Confusion	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Gastrointestinal disorders Constipation	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Gastrointestinal disorders Diarrhea	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
General disorders Fatigue	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Nervous system disorders Headache	8.8% 3/34 • 8 years used clinicaltrials.gov definitions
Investigations Fatigue	14.7% 5/34 • 8 years used clinicaltrials.gov definitions
Metabolism and nutrition disorders Hyperkalemia	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Metabolism and nutrition disorders Hyponatremia	11.8% 4/34 • 8 years used clinicaltrials.gov definitions
Metabolism and nutrition disorders Hypophosphatemia	11.8% 4/34 • 8 years used clinicaltrials.gov definitions
Psychiatric disorders Insomnia	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
General disorders Localized Edema	8.8% 3/34 • 8 years used clinicaltrials.gov definitions
Skin and subcutaneous tissue disorders Rash Acneiform	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
Nervous system disorders Seizure	8.8% 3/34 • 8 years used clinicaltrials.gov definitions
Blood and lymphatic system disorders Thrombocytopenia	5.9% 2/34 • 8 years used clinicaltrials.gov definitions
General disorders Pain	14.7% 5/34 • 8 years used clinicaltrials.gov definitions

Additional Information

Lara Rose

UCSD - Moores Cancer Center

Phone: 858-822-6575

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place