Summary

Eligibility
for people ages 0-18 (full criteria)
Location
at UCSF
Dates
study started
completion around
Principal Investigator
by Paul Harmatz, MD (ucsf)
Headshot of Paul Harmatz
Paul Harmatz

Description

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). Participants with MPS IIIA will be enrolled in two planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.

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)

Keywords

Mucopolysaccharidosis Type IIIA, Sanfilippo Syndrome, MPS IIIA, Mucopolysaccharidoses, Mucopolysaccharidosis III, DNL126

Eligibility

Locations

  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • Baylor College of Medicine and Texas Children's Hospita accepting new patients
    Houston Texas 77030 United States
  • University of Iowa Stead Family Children's Hospital accepting new patients
    Iowa City Iowa 52242 United States
  • University of North Carolina at Chapel Hill accepting new patients
    Chapel Hill North Carolina 27514 United States

Lead Scientist at University of California Health

  • Paul Harmatz, MD (ucsf)
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Denali Therapeutics Inc.
ID
NCT06181136
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 20 study participants
Last Updated