DYNE-251 for Duchenne Muscular Dystrophy

(DELIVER Trial)

Recruiting at 34 trial locations

Overseen By Dyne Clinical Trials

Age: < 18

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: Dyne Therapeutics

Must be taking: Glucocorticoids

Must not be taking: Eteplirsen, Gene therapy

Disqualifiers: Congestive heart failure, Major surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 60

86 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires participants to stay on a stable dose of glucocorticoids (a type of steroid medication) for at least 12 weeks before starting the study and to maintain this stable dose during certain periods of the study. If you are taking eteplirsen or similar exon-skipping therapies, you must stop them at least 12 weeks before joining the trial.

What is the purpose of this trial?

This trial tests DYNE-251, an IV drug, in people with Duchenne muscular dystrophy who can benefit from exon 51 skipping. The drug aims to help their muscles by increasing the dystrophin protein. It works by skipping a faulty part of their gene to produce more of this important protein. Eteplirsen (Exondys 51) was the first drug approved for exon 51 skipping in Duchenne muscular dystrophy.

Eligibility Criteria

Boys aged 4-16 with Duchenne muscular dystrophy (DMD) that can be treated by skipping exon 51, able to undergo muscle biopsy. They must have been on stable glucocorticoids for at least 12 weeks and have a healthy heart function. Non-ambulatory participants should not have lost mobility for more than two years.

Inclusion Criteria

I am between 4 and 16 years old.

A muscle in my arm can be safely biopsied.

I can lift my arms above my head without help.

Exclusion Criteria

I have received gene therapy before.

I haven't taken any experimental drugs that don't target gene skipping in the last 4 months.

I do not have uncontrolled symptoms of heart failure.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Multiple-Ascending Dose (MAD) / Placebo-Controlled Period

Participants receive either DYNE-251 or placebo every 4 or 8 weeks to assess safety, tolerability, and dystrophin protein levels

24 weeks

6 visits (in-person) or 3 visits (in-person) depending on dosing schedule

Open-Label Period

Participants receive DYNE-251 every 4 or 8 weeks to further assess safety and efficacy

24 weeks

6 visits (in-person) or 3 visits (in-person) depending on dosing schedule

Long-Term Extension (LTE) Period

Participants continue to receive DYNE-251 every 4 or 8 weeks for long-term safety and efficacy assessment

192 weeks

48 visits (in-person) or 24 visits (in-person) depending on dosing schedule

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• DYNE-251
• Placebo

Trial Overview The trial is testing DYNE-251's effects on safety, tolerability, and muscle protein levels in DMD patients compared to a placebo over multiple stages: an initial controlled phase of 24 weeks, followed by an open-label period of the same duration and then a long-term extension for up to 96 weeks.

Participant Groups

Group I: Placebo-Controlled MAD Period - Placebo Experimental Treatment 1 Intervention

Placebo will be administered Q4W or Q8W over 24 weeks.

Group II: Placebo-Controlled MAD Period - DYNE-251 Experimental Treatment 1 Intervention

DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.

Group III: Open-Label and Long-Term Extension Period - DYNE-251 Experimental Treatment 1 Intervention

DYNE-251 will be administered Q4W or Q8W for up to 192 weeks after participants complete the Placebo-Controlled MAD Period of the study.

Who Is Running the Clinical Trial?

Dyne Therapeutics

Lead Sponsor

Trials

3

Recruited

640+