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
Public ClinicalTrials.gov record NCT03952637. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Study identification
- NCT ID
- NCT03952637
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Enrollment
- 54 participants
Conditions and interventions
Conditions
Interventions
- AAV9-GLB1 Biological
- Abdominal ultrasound Procedure
- Audiology assessment with ABR Diagnostic Test
- Bone density scan (DEXA) Diagnostic Test
- Brain MRI/MRS/fMRI Procedure
- Echocardiogram Diagnostic Test
- Electrocardiogram (EKG) Diagnostic Test
- Electroencephalogram (EEG) awake and extended overnight Other
- Laboratory tests Diagnostic Test
- Lumbar puncture Procedure
- Methylprednisolone Drug
- Neurocognitive testing Behavioral
- Neurology exam Other
- Ophthalmology exam Procedure
- PICC or other Central line placement Behavioral
- Prednisone Drug
- Rituximab Drug
- Sirolimus Drug
- Skeletal survey Procedure
- Skin biopsy Procedure
- Speech and modified barium swallow study Procedure
Biological · Procedure · Diagnostic Test + 3 more
Eligibility (public fields only)
- Age range
- 6 Months to 12 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 18, 2019
- Primary completion
- Dec 31, 2027
- Completion
- Dec 31, 2027
- Last update posted
- Dec 10, 2025
2019 – 2028
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03952637, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 10, 2025 · Synced May 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03952637 live on ClinicalTrials.gov.