DNL126 Enzyme Replacement | Type A | Phase I-II | Denali

December 13, 2023

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

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