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NCT03952637 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study of Lysosomal Diseases and Gangliosidosis, sponsored by National Human Genome Research Institute (NHGRI).
- Recruiting
- Registry status
- Phase 1
- Development phase
- 54
- Enrollment target
- 1
- Study location
NCT03952637: Recruiting Phase 1 study of Lysosomal Diseases and Gangliosidosis, sponsored by National Human Genome Research Institute (NHGRI).
NCT03952637 is a Phase 1 study of Lysosomal Diseases and Gangliosidosis that is actively recruiting participants, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 54 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (10% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03952637, a Phase 1 study of Lysosomal Diseases and Gangliosidosis, is actively recruiting participants, sponsored by National Human Genome Research Institute (NHGRI).
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 54 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms. Objective: To test if a gene therapy helps Type I and Type II GM1 gangliosidosis symptoms. Eligibility: Type I subjects will be male and female \>= 6 months \<= 12 months of age at the time of full ICF signing. Type II subjects will be male and female \> 12 months old and \< 12 years old at the time of full ICF signing. Design: Participants will be screened with their medical history and a phone survey. Participants will stay at NIH for 8-10 weeks. Participants will have baseline tests: Blood, urine, and heart tests Hearing tests Ultrasound of abdomen EEG: Sticky patches on the participant s head will measure brain function. Lumbar puncture: A needle will be stuck into the participant s spine to remove fluid. MRI scans, bone x-rays, and bone scans: Participants will lie in a machine that takes pictures of the body IQ tests Neurology exams Central line placement Skin biopsy: A small piece of the participant s skin will be removed. Speech tests Participants will have an x-ray while swallowing food. Participants will take drugs by mouth and IV. This will get their immune system ready for therapy. Participants will get the gene therapy by IV. They may stay at NIH for a week to watch for side effects. Participants will have visits 3 and 6 months after treatment. Then visits will be every 6 months for 2 years. Then they will have a visit at 3 years. Visits will take 4-5 days. Participants will return to NIH once a year for 2 years for tests in an extension study....
Primary Outcome
Assess the safety of the AAV9/GLB1 vector (AAV9-GLB1) following intravenous delivery.
Conditions Studied
Interventions
- DRUG Sirolimus
- DRUG Rituximab
- DRUG Methylprednisolone
- BIOLOGICAL AAV9-GLB1
- PROCEDURE Abdominal ultrasound
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2019-08-19 |
| Est. Completion | 2028-01-01 |
| Phase | Phase 1 |
What NCT03952637 shows while recruiting
NCT03952637 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 54 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (10% lower).
The record links to 3 conditions, with Lysosomal Diseases appearing as the primary indexed condition, and to 5 interventions - of which Sirolimus is the first listed.
NCT03952637 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT03952637 about?
NCT03952637 is a clinical study titled "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". Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms. Objective: To test if a gene therapy helps Type I and ...
What is the current status of trial NCT03952637?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 54 participants. The study started on 2019-08-19. Estimated completion is 2028-01-01.
What conditions does trial NCT03952637 study?
This clinical trial studies the following conditions: Lysosomal Diseases, Gangliosidosis, GM1.
What interventions are being tested in trial NCT03952637?
The interventions under investigation include: Sirolimus (DRUG), Rituximab (DRUG), Methylprednisolone (DRUG), AAV9-GLB1 (BIOLOGICAL), Abdominal ultrasound (PROCEDURE).
Who is sponsoring clinical trial NCT03952637?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03952637 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03952637's enrollment target sits among peer trials
54 931st of 2000 higher than 1,053 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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