Page last updated: February 19, 2024
Page reviewed by: Dr. Cara O’Neill, FAAP
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Clinical Trial Summary
Study: “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)” ClinicalTrials.gov ID NCT06181136
Trial Listing: Read this clinical trial’s information on ClinicalTrials.gov, for more details including contact information to reach out to the study sites, full inclusion and exclusion criteria. We encourage you to check the Clinicaltrials.gov link frequently for updates on possible new study sites and changes in enrollment status or criteria.
Study opened: December 2023
Study design: Multicenter, open-label
Study status: Recruiting
Patients in the first study cohort A1 have been identified and are undergoing screening.
Route of administration: Intravenous through a central venous port
Study Length: The core study period is 25 weeks (approximately 6 months) and is followed by a 72-week (approximately 18 month) open-label extension (OLE).
Brief summary of DNL-126
DNL-126 is an investigational enzyme replacement therapy for patients with Sanfilippo syndrome type A. The enzyme deficient in Sanfilippo type A, SGSH, has been engineered to cross the blood brain barrier by binding to the transferrin receptor on blood vessels. It is intended to treat disease pathology in the brain and body.
This is the first study trialing the use of DNL-126 in humans.
Press Releases, web articles, presentations regarding DNL-126
- “Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases”, January 2023
- In the above press release, Denali stated that it expects to have “biomarker proof of concept and safety data from the Phase 1/2 study by the end of 2024.” Additionally, it expects to present preclinical data at WORLDSymposium 2024 in February 2024 and initiate dosing in Phase 1/2 in early 2024.
- In August 2023, Jonathan Gall and Kim Ramsey of Denali Therapeutics presented to the community during Cure Sanfilippo Foundation’s ADVANCE 2023 conference. They updated participants on Denali’s mission, preclinical work on DNL-126 and plans for the upcoming enzyme replacement clinical trial for Sanfilippo type A. Their presentation can be viewed below.