Pazopanib Hydrochloride in Treating Patients With Von Hippel-Lindau Syndrome
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|ClinicalTrials.gov Identifier: NCT01436227|
Recruitment Status : Active, not recruiting
First Posted : September 19, 2011
Last Update Posted : October 18, 2021
|Condition or disease||Intervention/treatment||Phase|
|Von Hippel-Lindau Syndrome||Other: Laboratory Biomarker Analysis Drug: Pazopanib Hydrochloride||Phase 2|
I. Evaluate safety and efficacy of treatment with pazopanib hydrochloride (pazopanib) for 6 months in patients with von Hippel-Lindau syndrome (VHL) who have a measurable VHL related lesion.
I. Evaluate rate of growth over time in target lesions before and after pazopanib treatment.
II. Evaluate need for surgical intervention over time in patients who receive pazopanib and compare to rate prior to receipt of drug.
III. Create an annotated tissue resource from patients with VHL for use in future research related to cancer.
I. Evaluate circulating factors in patients with VHL undergoing treatment with pazopanib.
II. Evaluate relationship between VHL genotype and response to pazopanib.
Patients receive pazopanib hydrochloride orally (PO) once daily (QD) on days 1-28. Treatment repeats every 4 weeks for up to 24 weeks in the absence of disease progression or unacceptable toxicity. Patients benefitting from treatment may continue pazopanib hydrochloride in the absence of disease progression.
After completion of study treatment, patients are followed up at 30 days and then every 3 months for up to 24 weeks.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of Pazopanib in Von Hippel-Lindau Syndrome|
|Actual Study Start Date :||January 17, 2012|
|Estimated Primary Completion Date :||April 11, 2022|
|Estimated Study Completion Date :||April 11, 2022|
Experimental: Treatment (pazopanib hydrochloride)
Patients receive pazopanib hydrochloride PO QD on days 1-28. Treatment repeats every 4 weeks for up to 24 weeks in the absence of disease progression or unacceptable toxicity. Patients benefitting from treatment may continue pazopanib hydrochloride in the absence of disease progression.
Other: Laboratory Biomarker Analysis
Drug: Pazopanib Hydrochloride
- Overall response rate (complete response + partial response) [ Time Frame: At 24 weeks ]Determined by the Response Evaluation Criteria in Solid Tumors. Estimated with its corresponding 95% posterior credible interval.
- Progressive disease rate [ Time Frame: Up to 24 weeks ]
- Drug discontinuation due to toxicity [ Time Frame: Up to 24 weeks ]
- Time to progression (TTP) [ Time Frame: Up to 24 weeks ]TTP will be estimated using the Kaplan-Meier method. Log-rank test will be performed to test the difference in survival between prognostic groups. Regression analyses of survival data based on the Cox proportional hazards model will be conducted on TTP. The proportional hazards assumption will be evaluated graphically and analytically, and regression diagnostics (e.g., martingale and Shoenfeld residuals) will be examined to ensure that the models are appropriate.
- Genetic mutations, discrete markers, and continuous biomarkers [ Time Frame: Up to 24 weeks ]Summary statistics, including frequency tabulation, means, standard deviations, median, and range, will be used to describe subject characteristics and marker data. The chi-squared test or Fisher's exact test will be used to test the association between two categorical variables, such as response and prognostic factors and discrete markers. The association among various continuous and discrete markers and response may be assessed first by the exploratory data analysis graphically and tested using Wilcoxon rank sum test.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01436227
|United States, Texas|
|M D Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Eric Jonasch||M.D. Anderson Cancer Center|