A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Summary
The registered summary isn't in our current data snapshot. Read it on the ClinicalTrials.gov record ↗. Why? →
Investigators
The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.
- Cynthia J Tifft, M.D.
National Human Genome Research Institute (NHGRI)
Principal Investigator
A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.
Sites
1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
Recruiting
Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.
Drugs studied
No drug or biological interventions are in our data for this study — it may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →
Registration & key dates
Registry attributes as recorded on the ClinicalTrials.gov study record.
- Start
- 19 Aug 2019
- Primary completion
- 1 Jan 2028
- Completion
- 1 Jan 2028
- Last update posted
- 11 Dec 2025
- Registered enrollment
- 54
- Registry id
- NCT03952637
Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 12 Jul 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.