Trial · NCT03952637
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
NCT03952637, Phase 1, Phase 2, Recruiting, National Human Genome Research Institute (NHGRI)
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. 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. Trials, record NCT03952637. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03952637
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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
- NCT number
- NCT03952637
- Status
- Recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 54 participants, estimated
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Interventions
AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
- First posted
- 2019-05-16
- Last updated
- 2026-09-21
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety | Several time points over 3 years | Assess the safety of the AAV9/GLB1 vector (AAV9-GLB1) following intravenous delivery. |
| Secondary | Brain MRI/MRS/fMRI | Varies between Type II and Type I subjects. | Type II subjects - Assess brain volume using a 3T MRI, and central nervous system (CNS) metabolite levels by magnetic resonance spectroscopy (MRI) between pre- and post-treatment and compared to historical controls |
| Secondary | CGI scale | Several timepoints over 3 years | Type II subjects - Assess change in disease severity using the Clinical Global Impressions (CGI) scale. |
| Secondary | Developmental changes | Varies between Type II and Type I subjects. | Type II subjects: Assess developmental change in the Growth Scale Scores on the Vineland Adaptive Behavior Scales Third Edition (Vineland-3) instrument between pre- and post-treatment. Type I subjects - Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) Composite score change from baseline |
| Secondary | Motor function | Varies between Type II and Type I subjects. | Type II subjects - Assess motor function using mobility scales developed as part of the natural history study |
| Secondary | Neurological function | Several timepoints over 3 years | Type I subjects - Neurological functional and developmental change from baseline |
| Secondary | Overall survival | Throughout study | Type I subjects life span is under 5 years |
| Secondary | Diffusion tensor imaging | Comparing timepoints throughout the study. Varies between Type II and Type I subjects. | Change in differential tractography using diffusion tensor imaging (DTI) pre- and post-treatment |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03952637
GET https://conductscience.org/api/trials/v1/NCT03952637/registered