Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Mucopolysaccharidosis Type IIIA Clinical Trial

Official title:
A Phase 1/2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Status Recruiting

Clinical Trial 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).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06181136
Study type	Interventional
Source	Denali Therapeutics Inc.
Contact	Clinical Trials at Denali Therapeutics
Phone	Email
Email	clinical-trials@dnli.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	December 7, 2023
Completion date	August 2028

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See also
Status	Clinical Trial	Phase
Active, not recruiting	`NCT04201405` - Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA	Phase 1/Phase 2
Completed	`NCT02037880` - Natural History Studies of Mucopolysaccharidosis III	N/A
Active, not recruiting	`NCT03612869` - Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)	Phase 2/Phase 3