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PTC299 for Treatment of Neurofibromatosis Type 2

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00911248
Recruitment Status : Terminated
First Posted : June 1, 2009
Last Update Posted : April 12, 2019
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
PTC Therapeutics

Tracking Information
First Submitted Date  ICMJE May 28, 2009
First Posted Date  ICMJE June 1, 2009
Last Update Posted Date April 12, 2019
Actual Study Start Date  ICMJE July 31, 2009
Actual Primary Completion Date March 31, 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 29, 2009)
To assess the effects of PTC299 on tumor volume and/or word recognition in patients with NF2. [ Time Frame: 48 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00911248 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 30, 2009)
  • To assess the effects of PTC299 on pure tone thresholds, brainstem auditory evoked responses (BAERs), and otoacoustic emissions (OAEs) in patients with NF2 [ Time Frame: 48 weeks ]
  • To determine if PTC299 alters the perception of tinnitus [ Time Frame: 48 weeks ]
  • To evaluate the effects of PTC299 on tumor blood flow [ Time Frame: 48 weeks ]
  • To assess the effects of PTC299 on concentrations of circulating angiogenic factors or cytokines [ Time Frame: 48 weeks ]
  • To describe the PTC299 safety profile [ Time Frame: 48 weeks ]
  • To evaluate compliance with PTC299 treatment [ Time Frame: 48 weeks ]
  • To assess PTC299 plasma exposure over time [ Time Frame: 48 weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 29, 2009)
  • To assess the effect of PTC299 on pure tone thresholds and brainstem auditory evoked responses (BAERs) and otoacoustic emissions (OAEs) in patients with NF2 [ Time Frame: 48 months ]
  • To determine if PTC299 alters the perception of tinnitus [ Time Frame: 48 months ]
  • To evaluate the effects of PTC299 on tumor blood flow [ Time Frame: 48 weeks ]
  • To assess the effects of PTC299 on concentrations of circulating angiogenic factors or cytokines [ Time Frame: 48 weeks ]
  • To describe the PTC299 safety profile [ Time Frame: 48 weeks ]
  • To evaluate compliance with PTC299 treatment [ Time Frame: 48 weeks ]
  • To assess PTC299 plasma exposure over time [ Time Frame: 48 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE PTC299 for Treatment of Neurofibromatosis Type 2
Official Title  ICMJE A Phase 2 Study to Assess the Efficacy, Safety, and Pharmacodynamic Activity of PTC299 in Patients With Neurofibromatosis Type 2
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) 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.
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 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Neurofibromatosis 2
Intervention  ICMJE Drug: PTC299
PTC299 will be administered orally at 100 mg/dose twice per day for up to 1 year or until tumor progression
Study Arms  ICMJE Experimental: PTC299
PTC299 administered at 100 mg/dose twice per day
Intervention: Drug: PTC299
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: August 24, 2018)
11
Original Estimated Enrollment  ICMJE
 (submitted: May 29, 2009)
25
Actual Study Completion Date  ICMJE March 31, 2012
Actual Primary Completion Date March 31, 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00911248
Other Study ID Numbers  ICMJE PTC299-ONC-007-NF2
NF080100
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party PTC Therapeutics
Study Sponsor  ICMJE PTC Therapeutics
Collaborators  ICMJE United States Department of Defense
Investigators  ICMJE
Principal Investigator: Jay Barth, MD PTC Therapeutics
PRS Account PTC Therapeutics
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP