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PTC299 for Treatment of Neurofibromatosis Type 2

NCT00911248 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2019-04-12

No results posted yet for this study

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) 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.

Conditions

  • Neurofibromatosis 2

Interventions

DRUG

PTC299

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

Sponsors & Collaborators

  • United States Department of Defense

    collaborator FED

  • PTC Therapeutics

    lead INDUSTRY

Principal Investigators

  • Jay Barth, MD · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-07-31
Primary Completion
2012-03-31
Completion
2012-03-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00911248 on ClinicalTrials.gov