Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants with DMD
Research type
Research Study
Full title
A Multicenter, Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants with Duchenne Muscular Dystrophy
IRAS ID
1013133
Contact name
Shelley Mendenhall
Contact email
Sponsor organisation
Keros Therapeutics, Inc.
Research summary
Duchenne muscular dystrophy (DMD) is an inherited disorder that causes gradual muscle loss. DMD mainly affects boys, with symptoms showing by 2 to 3 years of age and can cause premature death. There is currently no cure for DMD. Keros Therapeutics is sponsoring this trial to study an experimental treatment called KER-065 for DMD.
The main goal of the trial is to learn about the safety and tolerability of KER-065. To do this, researchers will study any new or worsening medical problems that participants have during and after treatment, including possible side effects.
This trial will include male patients at least 9 years old with DMD. People with certain medical conditions or who need to take certain medications may not be able to participate.
Participants will receive KER-065 as a subcutaneous (just under the skin) injection once every 4 weeks. Participants will continue to receive their standard DMD therapy at a stable regimen.
Participants will be in the trial for approximately 2 years, which will include the following:
-Screening (to check participants’ eligibility for the trial): up to 6 weeks
-Treatment period: up to 96 weeks, including the end of treatment visit (EOT) 4 weeks after their last injection
-Follow-up health checks: 4 weeks after the EOT.
The trial will be conducted at approximately 19 sites (hospitals, universities, and clinics) in multiple countries. A minimum of 6 sites will be located in the United Kingdom.
Participants will visit a trial site at least 33 times (some visits may be done at home). During site visits, trial doctors will collect blood and urine samples from participants, perform physical examinations, check their heart health, perform body imaging scans, and assess the status of their DMD, among others.
Participants may or may not receive any benefits from this trial. However, this trial will help researchers understand more about KER-065 and DMD.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
25/SC/0402
Date of REC Opinion
19 Jan 2026
REC opinion
Further Information Favourable Opinion