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 is a Phase 1 study that has completed, run by Nationwide Children's Hospital. The registered enrollment target is 6 participants.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00494195
The ClinicalTrials.gov registry entry for NCT00494195 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Nationwide Children's Hospital, which has 188 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which rAAV1.tMCK.human-alpha-sarcoglycan- First cohort is the first listed.
NCT00494195 reports 0 study locations.
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
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.