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

Phase 2
18 Years
Not Enrolling
Neurofibromatosis 2

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Trial Information

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

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.

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)

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess the effects of PTC299 on tumor volume and/or word recognition in patients with NF2.

Outcome Time Frame:

48 weeks

Safety Issue:


Principal Investigator

Jay Barth, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

PTC Therapeutics


United States: Food and Drug Administration

Study ID:




Start Date:

July 2009

Completion Date:

May 2012

Related Keywords:

  • Neurofibromatosis 2
  • Angiogenesis
  • Neurofibromatosis
  • Post-transcriptional control
  • PTC299
  • VEGF
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica
  • Neurofibromatosis 2



Massachusetts General HospitalBoston, Massachusetts  02114-2617