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MPS IIIA Disease Monitoring Program

StatusNot Yet Recruiting
PhaseNot specified
Started
View on ClinicalTrials.gov ↗

Record History

  1. 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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.
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Trial Details
NCT Number NCT07855562
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Enrollment 55 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov