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NCT04571970 · ClinicalTrials.gov registry record · Phase 1
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
A Phase 1 study, sponsored by REGENXBIO.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT04571970: Completed Phase 1 study, sponsored by REGENXBIO.
NCT04571970 is a Phase 1 study that has completed, run by REGENXBIO. 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
NCT04571970, a Phase 1 study, has completed, sponsored by REGENXBIO.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
Study Summary
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.
Primary Outcome
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 5.0)
Interventions
- GENETIC RGX-121
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2021-03-11 |
| Est. Completion | 2024-05-23 |
| Phase | Phase 1 |
What the finished NCT04571970 record still lists
NCT04571970 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 1 intervention - of which RGX-121 is the first listed.
NCT04571970 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04571970 about?
NCT04571970 is a clinical study titled "RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)". RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who ...
What is the current status of trial NCT04571970?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2021-03-11. Estimated completion is 2024-05-23.
What interventions are being tested in trial NCT04571970?
The interventions under investigation include: RGX-121 (GENETIC).
Who is sponsoring clinical trial NCT04571970?
This trial is sponsored by REGENXBIO, which has 9 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04571970'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
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