Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Status:
Recruiting
Trial end date:
2028-08-01
Target enrollment:
Participant gender:
Summary
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability,
pharmacokinetics (PK), pharmacodynamics (PD) and exploratory clinical efficacy of DNL126 in
participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks
(approximately 6 months) and is followed by a 72-week (approximately 18 month) open-label
extension (OLE).