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
Conditions
- Lysosomal Diseases
- Gangliosidosis
- GM1
Interventions
- BIOLOGICAL: AAV9-GLB1
- PROCEDURE: Abdominal ultrasound
- DRUG: Rituximab
- DRUG: Sirolimus
- DRUG: Methylprednisolone
- DRUG: Prednisone
- DIAGNOSTIC_TEST: Audiology assessment with ABR
- DIAGNOSTIC_TEST: Bone density scan (DEXA)
- DIAGNOSTIC_TEST: Electrocardiogram (EKG)
- DIAGNOSTIC_TEST: Echocardiogram
- OTHER: Electroencephalogram (EEG) awake and extended overnight
- DIAGNOSTIC_TEST: Laboratory tests
- PROCEDURE: Lumbar puncture
- PROCEDURE: Brain MRI/MRS/fMRI
- BEHAVIORAL: Neurocognitive testing
- OTHER: Neurology exam
- BEHAVIORAL: PICC or other Central line placement
- PROCEDURE: Skeletal survey
- PROCEDURE: Skin biopsy
- PROCEDURE: Speech and modified barium swallow study
Sponsor
National Human Genome Research Institute (NHGRI)
Collaborators