Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT04798235 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

See every version on ClinicalTrials.gov ↗

  1. Primary completion date: Mar 12, 2023 to Mar 12, 2027v7
  2. Trial status: Suspended to Active, Not Recruiting
    • Enrollment target: estimated 6 to confirmed 3
    v6
  3. Trial status: Active, Not Recruiting to Suspended Sponsor's reason: Regulatory information being askedv5
  4. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v4
  5. Eligibility criteria revisedv3
  6. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 1
    • Secondary endpoints revised (11)
    v2
  7. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.
Trial Details
NCT Number NCT04798235
Lead Sponsor Dr. Anupam Sehgal
Collaborators: Taysha Gene Therapies, Inc., GlycoNet
Conditions
(from sponsor)		 Infantile GM2 Gangliosidosis (Disorder)
Related terms Gangliosidoses, GM2, Sandhoff Disease, Tay-Sachs Disease
Enrollment 3 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov