Status of enrollment
Accepting new patients
Ages Eligible for Study
Type 1 (Early Onset Infantile) GM1 participants must be ≥4 months and <24 months of age at enrollment for Cohorts 3 and 4, and ≥1 month and <12 months of age at enrollment for Cohort 6 with onset of signs or symptoms of GM1 before 6 months of age. Type 2a (Late Onset Infantile) GM1 participants must be ≥6 months and <36 months of age at enrollment for Cohorts 1 and 2 and ≥6 months and ≤24 months of age at enrollment for Cohort 5 with onset of signs or symptoms of GM1 between 6 and 18 months of age.
Genders Eligible for Study
Females and males
Disease indication
GM1 Gangliosidosis
Principal Investigator
Caroline Hastings, MD
Contact
Caroline Hastings, MD
Additional study eligibility details can be found at UCSF Clinical Trials.