- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Interventions
- PBGM01
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 24 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 4
- States / cities
- Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
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- Conditions
- GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
- Interventions
- miglustat, Ketogenic Diet
- Drug · Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Up to 204 Months
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- GM1 Gangliosidosis
- Interventions
- Not listed
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- Not listed
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 2
- States / cities
- Oakland, California • Philadelphia, Pennsylvania