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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

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Trial Profile

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
Phase of Trial: Phase I/II

Latest Information Update: 13 Jul 2025

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At a glance

  • Drugs DNL 126 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Biomarker; Proof of concept; Registrational; Therapeutic Use
  • Sponsors Denali Therapeutics Inc

Most Recent Events

  • 14 May 2025 Planned number of patients changed from 20 to 26, Primary endpoints has been amended and trial focus has been shifted to TU.
  • 06 Nov 2024 According to a Denali therapeutics Inc media release, Based on preliminary data and a positive regulatory environment, company have recently expanded the study to support a potential accelerated path.
  • 06 Nov 2024 Results published in the Media Release

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