Astellas

This trial is not actively recruiting.

This trial has completed recruitment on this platform, and is no longer accepting new referrals.

Astellas Phase 1b Study of ASP0367 in pediatric male patients with Duchenne Muscular Dystrophy (DMD)

A study to assess the safety, tolerability and preliminary efficacy of ASP0367(MA-0211) in pediatric male participants with Duchenne muscular dystrophy (DMD)

Trial ID: NCT04184882

Trial Details

We are testing ASP0367, an investigational drug, to find out if it relieves key Duchenne Muscular Dystrophy (DMD) symptoms. ASP0367 is designed to increase the number and function of the mitochondria in cells, thereby increasing energy production. This has the potential to improve muscle health and function, reduce inflammation, and increase endurance in people living with DMD.

A Phase 1b clinical trial is open now in the United States. The primary purpose of the study is to test the safety and side effects of ASP0367, and to learn how the body processes it. The study is also looking at how ASP0367 affects muscle function (e.g. using an arm cycling test). This study has four parts and takes up to 8 months to finish in total.

  1. Screening period for 4 weeks (where your son would do tests to see if the study is a match for him)
  2. Double-blind period for 12 weeks (where neither you nor the study doctor would not know whether your son was taking ASP0367 tablets or placebo tablets [A placebo looks like a drug or treatment but does not have an active product or treatment in it])
  3. Open-label period for 12 weeks (where everyone takes ASP0367)
  4. Follow up period for 4 weeks (where your son would not take the study medicine).

If you choose not to take part in the optional open-label period, the study will take 5 months to finish.

Estimated Enrollment

18 Participants

Phase

1b

Eligibility Criteria

Participants must:
  • Be a boy age 8 to 16 years old in the U.S.
  • Have a diagnosis of Duchenne Muscular Dystrophy (DMD)
  • Be regularly taking corticosteroid medications such as prednisolone or deflazacort
  • Have some trouble walking quickly
  • Can move arms up to the shoulder height
Participants must not:
  • Have a recent illness or have a serious infection, mental health condition, or behavioral problem, or is not healthy enough for the study
  • Have heart problems (ejection fraction <55%, long QT interval, high levels of cTnI on lab test)
  • Have used certain types of drugs within 4 weeks of the study
  • Have kidney or liver problems
  • Have recently taken or is currently taking any other investigational drug

What Will Happen in This Study

What’s Involved

The study takes up to 8 months to finish in total. If participants do not choose to take part in the open-label part, the study takes about 5 months to complete. Participants will take the tablet by mouth each day during the treatment period.

Participants will complete tests related to their health and condition at about seven hospital visits and eight home visits by a qualified home healthcare provider, as well as through follow-up phone calls. These tests may include a physical examination (height, weight, vital signs), 12-lead electrocardiogram (ECG), heart imaging (echocardiography), blood/urine test, assisted 6-minute arm cycling test, examination of arms, 2-minute walk test, breathing function tests, and participant questionnaires.

Travel support is available for clinical trial participants and their families.

Additional site locations will become available on a rolling basis. Please check back if you do not see an enrolling site near you.

You will need to bring your child to one of the study clinics taking part in this clinical trial.

Sites are located in the cities/states identified with the red pin:

Frequently Asked Questions (FAQs)