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Clinical trial

A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

NCT04360265 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT04360265
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Registry record

As filed in ClinicalTrials.gov.

Title
A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 3
Overall status
Enrolling by invitation
Start date
September 28, 2020
Completion date
August 31, 2027
Enrollment
41 (estimated)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

mps iiia, mucopolysaccharidosis iiia, sanfilippo a, sanfilippo syndrome