Pazopanib for Metastatic Alveolar Soft Part Sarcoma

Study Overview
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Brief Summary
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Alveolar soft part sarcoma (ASPS), a rare subset of STS (<1%) harbors t(X;17)(p11;q25) translocation and produces resultant ASPL-TFE3 fusion protein. Due to its nature of high expression of angiogenic factors, sunitinib and cediranib produced overall response rates of 55% and 43%, respectively. However, the efficacy of pazopanib is unknown in metastatic ASPS.

Detailed Description
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Pazopanib, a multi-targeted anti-angiogenesis inhibitor significantly prolonged progression-free survival (PFS) in patients with metastatic soft-tissue sarcoma (STS) after failure to anthracycline-based regimen (pazopanib vs placebo, 4.6 vs 1.6 months, HR=0.31, 95% CI 0.24-0.40; P < .0001). Regarding sunitinib (continuous daily dose of 37.5mg), after a median duration of 10 months, median OS and PFS were 19 months and 17 months, respectively in a small retrospective study (ASPS, N=9). With regard to cediranib, 6-month PFS was over 60%. In addition, randomized phase II trial of sunitinib vs cediranib with cross-over at disease progression was recently initiated (NCT01391962). However, the efficacy of pazopanib is unknown in metastatic ASPS.

Official Title
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A Phase II Trial of Pazopanib in Patients With Metastatic Alveolar Soft Part Sarcoma

Conditions
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Metastatic Alveolar Soft Part Sarcoma

Intervention / Treatment
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* Drug: Pazopanib


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Histologically confirmed diagnosis of alveolar soft part sarcoma harboring TFE3 fusion at stage IV or at relapse Age ≥ 18 years Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 Measurable lesion defined by RECIST v1.1 Chemo-naïve or prior chemotherapies Adequate organ function Exclusion Criteria: Prior malignancies Active CNS disease High-risk for gastrointestinal bleeding Significant cardiovascular disease Uncontrolled hypertension Bleeding diathesis

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pazopanib<br>Pazopanib 800mg po qd until disease progression | Drug: Pazopanib<br>* Pazopanib 800mg qd daily for 4 weeks = 1 cycle<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response rate (ORR) | ORR based on RECIST v1.1 | One year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events | Safety based on CTCAE v4.0 | One year | 
| Progression-free survival |  | 6 month | 
| Overall survival |  | Two years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pazopanib, Alveolar soft part sarcoma