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Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

StatusNot Yet Recruiting
PhasePhase 1
Started
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion moved at least 12 months later Jun 1, 2038 → Jun 1, 2039
See other at-risk trials from Tippi Mackenzie

Record History

See every version on ClinicalTrials.gov ↗

  1. Primary completion pushed from Jun 1, 2038 to Jun 1, 2039
    • Start date: Jun 1, 2026 to Jan 1, 2027
    v3
  2. Study title shortened, about 3 wordsv2
  3. 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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.
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
Trial Details
NCT Number NCT07479953
Lead Sponsor Tippi Mackenzie
Conditions
(from sponsor)		 GM1 Gangliosidoses, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2
Related terms Gangliosidosis, GM1
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov