RGX-111 Gene Therapy in Patients With MPS I
← catalyst calendarNCT03580083 · readout in 322 d
Sponsored by REGENXBIO Inc. (industry) · RGNX — their whole pipeline →.
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Why the sponsor stopped it
The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.
As filed on the registry record, in the sponsor's own words.
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2019-04-03 | actual | When the study began enrolling |
| Primary completion | 2027-07-07 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2029-03-06 | estimated | The whole study's end, after follow-up |
| First posted | 2018-07-09 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-12 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Mucopolysaccharidosis Type I (MPS I)
- Hurler Syndrome
- Hurler-Scheie Syndrome
Intervention
- Genetic: RGX-111
Primary outcome
what the primary-completion date above is the date OFPublications
- PMID 37624739 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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