Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00759876 · ClinicalTrials.gov registry record · Phase 2
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT00759876: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT00759876 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 36 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00759876, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study Summary
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DMD in approximately 10-15% of boys with the disease. Ataluren is an orally-delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a extension trial that will evaluate the long-term safety of ataluren in boys with nonsense mutation DMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, strength, and other important clinical and laboratory measures.
Primary Outcome
TEAE: any untoward medical occurrence or undesirable event(s) that begins or worsens following administration of the study drug, whether or not considered related to the treatment by the Investigator. Severity of an adverse event (AE) was classified as: mild (does not interfere with usual function), moderate (interferes with usual function; may require medical intervention), severe (interferes significantly with usual function; likely require medical intervention), life-threatening, and fatal. D
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2008-08-13 |
| Est. Completion | 2010-05-17 |
| Phase | Phase 2 |
Why NCT00759876 stopped before completion
NCT00759876 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT00759876 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00759876 about?
NCT00759876 is a clinical study titled "Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)". Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walkin...
What is the current status of trial NCT00759876?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2008-08-13. Estimated completion is 2010-05-17.
What interventions are being tested in trial NCT00759876?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT00759876?
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 NCT00759876's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00929006 · 36 participants · Early Phase 1
Acute Progesterone Suppression of Wake vs. Sleep Luteinizing Hormone Pulse Frequency in Pubertal Girls With and Without Hyperandrogenism
- NCT01891318 · 36 participants · Phase 1
Neoadjuvant Radiosurgery for Resectable Brain Metastases: Phase I/II Study
- NCT02291848 · 36 participants · Phase 1
Tumor-Associated Antigen-Specific Cytotoxic T-Lymphocytes for Multiple Myeloma
- NCT02366819 · 36 participants · Phase 4
Genetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI