Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00494195 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
A Phase 1 study, sponsored by Nationwide Children's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT00494195: Completed Phase 1 study, sponsored by Nationwide Children's Hospital.
NCT00494195 is a Phase 1 study that has completed, run by Nationwide Children's Hospital. The registered enrollment target is 6 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00494195, a Phase 1 study, has completed, sponsored by Nationwide Children's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
Study Summary
Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating children and adults with LGMD2D.
Interventions
- GENETIC rAAV1.tMCK.human-alpha-sarcoglycan- First cohort
- GENETIC Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2008-03 |
| Est. Completion | 2011-08 |
| Phase | Phase 1 |
What the finished NCT00494195 record still lists
NCT00494195 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 2 interventions - of which rAAV1.tMCK.human-alpha-sarcoglycan- First cohort is the first listed.
NCT00494195 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00494195 about?
NCT00494195 is a clinical study titled "Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)". Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating ...
What is the current status of trial NCT00494195?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2008-03. Estimated completion is 2011-08.
What interventions are being tested in trial NCT00494195?
The interventions under investigation include: rAAV1.tMCK.human-alpha-sarcoglycan- First cohort (GENETIC), Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort (GENETIC).
Who is sponsoring clinical trial NCT00494195?
This trial is sponsored by Nationwide Children's Hospital, which has 188 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00494195's enrollment target sits among peer trials
6 1974th of 2000 higher than 12 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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 registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia