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NCT03520751 · ClinicalTrials.gov registry record · Phase 1
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
A Phase 1 study, sponsored by Nationwide Children's Hospital.
- Not yet
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
NCT03520751: Clinical Trial Phase 1 study, sponsored by Nationwide Children's Hospital.
NCT03520751 is a Phase 1 study that has not yet begun recruiting, run by Nationwide Children's Hospital. The registered enrollment target is 3 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03520751, a Phase 1 study, has not yet begun recruiting, sponsored by Nationwide Children's Hospital.
- NOT YET RECRUITING
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
Study Summary
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.
Interventions
- DRUG scAAV1.tMCK.NTF3
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2027-04 |
| Est. Completion | 2030-04 |
| Phase | Phase 1 |
What is known before NCT03520751 opens enrollment
NCT03520751 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 1 intervention - of which scAAV1.tMCK.NTF3 is the first listed.
NCT03520751 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03520751 about?
NCT03520751 is a clinical study titled "Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A". This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.
What is the current status of trial NCT03520751?
This trial is currently not yet recruiting. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2027-04. Estimated completion is 2030-04.
What interventions are being tested in trial NCT03520751?
The interventions under investigation include: scAAV1.tMCK.NTF3 (DRUG).
Who is sponsoring clinical trial NCT03520751?
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 NCT03520751's enrollment target sits among peer trials
3 1999th of 2000 higher than 1 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
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