DYNE-251 for Duchenne Muscular Dystrophy

(DELIVER Trial)

Not currently recruiting at 37 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the safety and effectiveness of a new treatment called DYNE-251 for individuals with Duchenne muscular dystrophy (DMD) who have a specific gene mutation targetable by exon 51 skipping. The trial will assess how this treatment affects dystrophin protein levels in muscles, which are crucial for muscle function. Participants will initially receive either DYNE-251 or a placebo, and later, all will have the opportunity to receive DYNE-251 in an open-label phase. Boys aged 4 to 16 with a confirmed diagnosis of DMD and the specific gene mutation may qualify if they have been on stable glucocorticoid treatment for at least three months. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that DYNE-251 is likely to be safe for humans?

Research has shown that DYNE-251 is generally safe. In studies, patients who took DYNE-251 showed early signs of improved muscle function. Most importantly, the treatment was well-tolerated, with few serious side effects. While some side effects might occur, they were neither common nor severe. This makes DYNE-251 a promising option for those considering joining clinical trials for Duchenne muscular dystrophy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne muscular dystrophy?

DYNE-251 is unique because it's designed to treat Duchenne Muscular Dystrophy (DMD) by targeting the genetic cause of the disease. Unlike standard treatments such as corticosteroids, which mainly manage symptoms, DYNE-251 focuses on exon skipping, a process that helps produce functional dystrophin protein. This targeted approach could potentially slow down muscle degeneration more effectively. Researchers are excited because DYNE-251's mechanism might offer a longer-lasting solution compared to current symptom-focused therapies.

What evidence suggests that DYNE-251 might be an effective treatment for Duchenne muscular dystrophy?

Research has shown that DYNE-251 may help treat Duchenne muscular dystrophy (DMD). In one study, patients who received 20 mg/kg of DYNE-251 every four weeks had an average of 8.72% of normal dystrophin levels in their muscles. Dystrophin is crucial for muscle function, and higher levels can strengthen muscles. Over a year, patients reported noticeable improvements in their daily activities. The trial includes a placebo-controlled period where some participants will receive DYNE-251, while others will receive a placebo. This evidence suggests that DYNE-251 could effectively improve muscle function in DMD. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. investors.dyne-tx.com investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-long-term-clinical-data-phase-12

Dyne Therapeutics Announces New Long-Term Clinical ... At the 6-month time point, patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 8.72% of normal (adjusted ...

2. parentprojectmd.org parentprojectmd.org/dyne-therapeutics-inc-announced-today-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy/

Dyne Therapeutics, Inc. announced today FDA ... DYNE-251 is designed to enable the production of near full-length dystrophin, a key protein for muscle function. · Breakthrough Therapy ...

3. investors.dyne-tx.com investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-additional-one-year-clinical-data

Dyne Therapeutics Announces Additional One-Year ... Patient Reported Outcomes: Meaningful and sustained improvement from ... Duchenne muscular dystrophy (DMD) data with its FORCE platform.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05524883

Study Details | NCT05524883 | Safety, Tolerability, ... The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) ...

5. neurology.org neurology.org/doi/10.1212/WNL.0000000000211198

Safety and Efficacy of DYNE-251 in Males with DMD ... The mean corresponding muscle content-adjusted dystrophin levels were 7.64% and 8.72% of normal. DYNE-251 led to improvements across multiple functional ...

6. parentprojectmd.org parentprojectmd.org/dyne-therapeutics-announces-positive-new-clinical-data-from-phase-1-2-deliver-trial-of-dyne-251-in-duchenne/

Dyne Therapeutics Announces Positive New Clinical Data ... Patients treated with 20 mg/kg Q4W of DYNE-251 had a mean absolute dystrophin level of 3.71% of normal at 6 months. When adjusting for muscle ...