DNLI-J-0001

• Research type

  Research Study

• Full title

  A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease

• IRAS ID

  1013476

• Contact name

  Clinical Trials Group

• Contact email

  clinical-trials@dnli.com

• Sponsor organisation

  Denali Therapeutics Inc.

• Clinicaltrials.gov Identifier

  NCT07354724

• Research summary

  This is a phase I open-label study, funded by Denali Therapeutics Inc., which purpose is to learn more about a new investigational drug called DNL952 and how it works in people with Late-Onset Pompe Disease (LOPD).
  LOPD is a condition where the body cannot break down a kind of sugar called glycogen because a deficiency in an enzyme called acid alpha-glucosidase (GAA). This causes glycogen to build up the muscles. Over time, this can make the muscles weak, including the muscles used for walking and breathing. Glycogen may also build up in the brain, spinal cord, and nerves of the nervous system.
  DNL952 is a study drug that is made up of the GAA enzyme connected to a protein that helps GAA get into different parts of the body, including the muscle and the nervous system.
  The goals of this study are to understand:
  • If the study drug is safe and well tolerated
  • How much of the study drug people can safely receive
  • How the body processes the study drug (called pharmacokinetics or PK)
  • How the study drug affects levels of certain markers that reflect the effects of Pompe Disease on the body, including in the blood and urine (called pharmacodynamics)

  This is the first study of DNL952 in humans.
  In total, up to 32 adults with LOPD will take part in this study.
  This study has up to six (6) cohorts, two (2) of which are planned and four (4) of which are optional.
  Cohorts A1, A2, A3 and A4: These cohorts will enroll participants who are currently receiving treatment with either avalglucosidase alfa (also known as Nexviazyme®/Nexviadyme®) or cipaglucosidase alfa in combination with miglustat (also known as Pombiliti® + Opfolda®).
  Cohorts B1 and B2 (optional): If opened, these cohorts will enroll participants who have not been treated with enzyme replacement therapy.

  Participants will be involved in the study for approximately 1 year from screening through follow-up.

• REC name

  West Midlands - Edgbaston Research Ethics Committee

• REC reference

  26/WM/0031

• Date of REC Opinion

  18 Mar 2026

• REC opinion

  Further Information Favourable Opinion