PTC299 for Treatment of Neurofibromatosis Type 2

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-07-31

Study Completion Date

2012-03-31

Brief Summary

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Formation of new blood vessels (angiogenesis) is important for tumor growth in neurofibromatosis type 2 (NF2). It is known that tumors make a protein called vascular endothelial growth factor (VEGF) and there are higher levels of VEGF in the tumors and blood of many patients with NF2. VEGF stimulates the formation of blood vessels that supply the tumor with nutrients and oxygen. PTC299 is an oral drug that has been shown to decrease production of VEGF in animal models of human cancer. In these animal models, oral PTC299 administration decreases VEGF levels in the tumor and in the bloodstream, decreases blood vessel numbers in the tumor, and significantly slows or halts tumor growth. Safety studies in research animals indicate good tolerability at doses and drug levels that are higher than those planned for the clinical studies. Results from Phase 1a studies in healthy volunteers indicate that PTC299 achieves levels of PTC299 in the bloodstream that are known to be active in animal models of human tumor. This Phase 2 study is designed to test the hypothesis that PTC299 will be tolerable and will show evidence of VEGF reduction, antitumor activity, and hearing improvement when administered orally to patients with NF2.

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Detailed Description

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The study will be conducted in 2 stages. In Stage 1 of the study, 11 patients will receive daily treatment with PTC299 administered at 100 mg/dose twice per day for up to 1 year or until tumor progression. If no subject responds with tumor shrinkage or an improvement in hearing, then the study will be stopped. If ≥1 out of 11 subjects respond, then the study will proceed to Stage 2 to enroll an additional 14 subjects for a total of 25 subjects.

Conditions

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Neurofibromatosis 2

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PTC299

PTC299 administered at 100 mg/dose twice per day

Group Type EXPERIMENTAL

PTC299

Intervention Type DRUG

PTC299 will be administered orally at 100 mg/dose twice per day for up to 1 year or until tumor progression

Interventions

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PTC299

PTC299 will be administered orally at 100 mg/dose twice per day for up to 1 year or until tumor progression

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Age ≥18 years
* Diagnosis of NF2
* Presence of vestibular schwannomas
* Evidence of progressive increase in vestibular schwannoma size or worsening hearing loss due to vestibular schwannoma
* Adequate functional status (Karnofsky Performance Score ≥60)
* Adequate bone marrow, liver, kidney function
* If sexually active, willingness to use effective barrier or medical contraception
* For women of childbearing potential, no pregnancy or breast-feeding
* Discontinuation of other therapies (except corticosteroids) for the treatment of NF2 and resolution of any acute toxic effects of prior therapies
* Willingness and ability to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions
* Willingness to provide informed consent

Exclusion Criteria

* Uncontrolled hypertension, major bleeding, HIV infection, or recent acute cardiovascular event
* Prior exposure to another anti-angiogenic therapy (eg, bevacizumab, sunitinib)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No