PTC299 for Treatment of Neurofibromatosis Type 2

ClinicalTrials.gov ID: NCT00911248

Public ClinicalTrials.gov record NCT00911248. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Sep 9, 2026, 8:41 PM EDT

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Official title

A Phase 2 Study to Assess the Efficacy, Safety, and Pharmacodynamic Activity of PTC299 in Patients With Neurofibromatosis Type 2

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID: NCT00911248
Recruitment status: Terminated
Study type: Interventional
Phase: Phase 2
Lead sponsor: PTC Therapeutics; Industry
Enrollment: 11 participants

Conditions and interventions

Conditions

Neurofibromatosis 2

Interventions

PTC299 Drug

Drug

Eligibility (public fields only)

Age range: 18 Years and older
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Jul 30, 2009
Primary completion: Mar 30, 2012
Completion: Mar 30, 2012
Last update posted: Apr 11, 2019

2009 – 2012

United States locations

U.S. sites: 1
U.S. states: 1
U.S. cities: 1

Facility	City	State	ZIP	Site status
Massachusetts General Hospital	Boston	Massachusetts	02114	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT00911248, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Apr 11, 2019 · Synced Sep 9, 2026

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The complete protocol, eligibility criteria, and contact information for NCT00911248 live on ClinicalTrials.gov.

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