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
Status at this site: Recruiting
- 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
- Biological · Procedure · Drug + 3 more
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland