Status:
TERMINATED
PTC299 for Treatment of Neurofibromatosis Type 2
Lead Sponsor:
PTC Therapeutics
Collaborating Sponsors:
United States Department of Defense
Conditions:
Neurofibromatosis 2
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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) a...
Detailed Description
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 progres...
Eligibility Criteria
Inclusion
- 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
- Uncontrolled hypertension, major bleeding, HIV infection, or recent acute cardiovascular event
- Prior exposure to another anti-angiogenic therapy (eg, bevacizumab, sunitinib)
Key Trial Info
Start Date :
July 31 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 31 2012
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT00911248
Start Date
July 31 2009
End Date
March 31 2012
Last Update
April 12 2019
Active Locations (1)
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1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114