Trial Outcomes & Findings for Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow (NCT NCT00330564)

Results Overview

Safety evaluation = Number of participants with treatment terminating toxicity using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 3.0. Early stopping rules applied when treatment terminating toxicity occurred in the first 6 week cycle. Recurring grade 3 toxicity requires dose reduction, with no more than 2 dose reductions permitted. If no improvement after 4 weeks, patient is taken off drug and off study, and the event recorded as treatment terminating toxicity.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

12 weeks

Results posted on

2024-06-27

Participant Flow

Recruitment Period: 5/19/2006 to 3/17/2010. Participants recruited from routine appointments at a Comprehensive Cancer Center.

Study terminated early due to slow accrual.

Participant milestones

Participant milestones
Measure	SU011248 (Sutent, Sunitinib Malate) 50 mg/day orally for 4 weeks
Overall Study STARTED	15
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	SU011248 (Sutent, Sunitinib Malate) n=15 Participants 50 mg/day orally for 4 weeks
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	36 participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Intent to treat once the first dose was taken.

Safety evaluation = Number of participants with treatment terminating toxicity using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 3.0. Early stopping rules applied when treatment terminating toxicity occurred in the first 6 week cycle. Recurring grade 3 toxicity requires dose reduction, with no more than 2 dose reductions permitted. If no improvement after 4 weeks, patient is taken off drug and off study, and the event recorded as treatment terminating toxicity.

Outcome measures

Outcome measures
Measure	SU011248 (Sutent, Sunitinib Malate) n=15 Participants 50 mg/day orally for 4 weeks
Safety of Sunitinib Administration in Participants With Von Hippel-Lindau Syndrome (VHL)	15 participants

SECONDARY outcome

Timeframe: Baseline to 12 months (evaluations at 6 and 12 months)

Population: Secondary end point of efficacy showed response of renal cell carcinomas, which responded better to sunitinib therapy than other VHL related lesions using RECIST measure.

Response of VHL lesions (number) evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) of Complete Response (CR): Disappearance of all target lesions, and Partial Response (PR): At least a 30% decrease in the sum of longest diameter (LD) of target lesions, reference baseline sum LD. Progressive Disease (PD): 20% increase in LD sum and Stable Disease (SD): Insufficient shrinkage to qualify for PR nor increase to qualify for PD. Degree and timing of response in affected organs evaluated in order to determine organ specific kinetics of therapy.

Outcome measures

Outcome measures
Measure	SU011248 (Sutent, Sunitinib Malate) n=18 renal cell carcinoma lesions 50 mg/day orally for 4 weeks
Number of VHL Lesion Complete + Partial Responses CR	0 VHL lesion
Number of VHL Lesion Complete + Partial Responses PR	6 VHL lesion

Adverse Events

SU011248 (Sutent, Sunitinib Malate)

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	SU011248 (Sutent, Sunitinib Malate) n=15 participants at risk 50 mg/day orally for 4 weeks
Blood and lymphatic system disorders Neutropenia	26.7% 4/15 • Number of events 4 • 3 years, 8 months
General disorders fatigue	33.3% 5/15 • Number of events 5 • 3 years, 8 months
Skin and subcutaneous tissue disorders hand foot syndrome	13.3% 2/15 • Number of events 2 • 3 years, 8 months
Cardiac disorders hypertension	6.7% 1/15 • Number of events 1 • 3 years, 8 months
Gastrointestinal disorders nausea	13.3% 2/15 • Number of events 2 • 3 years, 8 months

Additional Information

Quality Assurance Specialist

UT MD Anderson Cancer Center

Phone: 713-563-1602

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place