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NCT00911248 · ClinicalTrials.gov registry record · Phase 2
PTC299 for Treatment of Neurofibromatosis Type 2
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 11
- Enrollment target
NCT00911248: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT00911248 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 11 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00911248, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 11 participants
- Enrollment target
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.
Interventions
- DRUG PTC299
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2009-07-31 |
| Est. Completion | 2012-03-31 |
| Phase | Phase 2 |
Why NCT00911248 stopped before completion
NCT00911248 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 11 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which PTC299 is the first listed.
NCT00911248 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00911248 about?
NCT00911248 is a clinical study titled "PTC299 for Treatment of Neurofibromatosis Type 2". 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 stimulate...
What is the current status of trial NCT00911248?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 11 participants. The study started on 2009-07-31. Estimated completion is 2012-03-31.
What interventions are being tested in trial NCT00911248?
The interventions under investigation include: PTC299 (DRUG).
Who is sponsoring clinical trial NCT00911248?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00911248's enrollment target sits among peer trials
11 1967th of 2000 higher than 33 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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