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Clinical trial

A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)

NCT07236606 is registered in ClinicalTrials.gov with REGENXBIO Inc. as lead sponsor.

NCT NCT07236606
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)
Lead sponsor
REGENXBIO Inc.
Phase
Phase 3
Overall status
Active, not recruiting
Start date
November 25, 2025
Completion date
May 30, 2031
Enrollment
2 (estimated)
Sites
1

Conditions

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

hunter syndrome (mps ii), mps ii