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Clinical trial

An Open-Label, Single Injection, Ascending Dose Phase I/II Clinical Study of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

NCT07818759 is registered in ClinicalTrials.gov with NeuroGT as lead sponsor.

NCT NCT07818759
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-Label, Single Injection, Ascending Dose Phase I/II Clinical Study of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
Lead sponsor
NeuroGT
Phase
Phase 1/2
Overall status
Not yet recruiting
Start date
January 18, 2027
Completion date
December 31, 2029
Enrollment
9 (estimated)
Sites
1

Conditions

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

sanfilippo syndrome (mps iii)