ARODM1-1001
Research type
Research Study
Full title
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who are ≥18 to ≤65 Years
IRAS ID
1012703
Contact name
Yvonne Nguyen
Contact email
Sponsor organisation
Arrowhead Pharmaceuticals, Inc.
Research summary
Type 1 myotonic dystrophy (DM1) is a degenerative neuromuscular disorder,characterized by changes in the DMPK gene and is associated with muscle weakness and wasting, and physical disability.Currently,no approved disease modifying therapy for DM1 is available.Treatments focus on symptom management (including physical therapy, exercise and use of orthoses or wheelchair).
This is a Phase 1/2a dose-escalating study involving adults between 18 and 65 years old with DM1, who meet the eligibility criteria.It consists of Part I (Group 1 and 2):Single Ascending Doses of ARO-DM1 or placebo administered intravenously, and Part II (Group 3,4 and 5):Multiple Ascending Doses of ARO-DM1 or placebo administered intravenously.In Part II there are also 3 optional groups (Groups 6, 7, and 8) that may receive either a single dose or multiple doses of ARO-DM1 or placebo subcutaneously. The anticipated number of participants to be included is 78 in 31 sites in 11 Countries. The duration of study participation for Part I will be up to 150 days. For Part 2: The duration of study will be up to 240 days for Groups 3, 4, and 5, and optional multiple dose Groups 6, 7, and 8.If the optional 10-week dosing interval is chosen for Groups 5, 6, 7, and 8, the duration will be up to 200 days. The duration of study participation will be up to 150 days for Optional single dose Groups 6, 7, and 8.Because this study is the first human exposure to the experimental therapeutic ARO-DM1,both clinical benefits and risks to participants are unknown.ARO-DM1 has the potential to halt, or slow, the progression of DM1.The active ingredient,contained within the ARO-DM1, is intended to treat patients with DM1and lead to improved muscle strength and function.The trial was designed to minimize burden to subjects while still obtaining necessary data to fulfill the trial objectives and focus on recruiting a subject population in whom ARO-DM1 will be safe and are in line with criteria from other recent DM1 studies.REC name
London - London Bridge Research Ethics Committee
REC reference
25/LO/0789
Date of REC Opinion
2 Dec 2025
REC opinion
Further Information Favourable Opinion