Zeleciment Basivarsen for Myotonic Dystrophy

Recruiting at 4 trial locations

Overseen By Dyne Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Dyne Therapeutics

Must not be taking: GLP-1 agonists

Disqualifiers: Congenital DM1, recent major surgery, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 9

150 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

Are You a Good Fit for This Trial?

Inclusion Criteria

Body mass index (BMI) less than 35 kilograms per meter square (kg/m^2)

I have DM1 confirmed by genetic testing with more than 100 repeats.

I can walk 10 meters and stand up from a chair five times on my own.

Exclusion Criteria

I have been diagnosed with congenital DM1.

I have not had major surgery in the last 12 weeks, except for a pacemaker or defibrillator.

I have not used GLP-1 medications like semaglutide recently.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 8 weeks

Placebo-Controlled Period

Participants are randomized to receive either DYNE-101 or placebo every 8 weeks for up to 48 weeks

48 weeks

6 visits (in-person)

Long-Term Extension Period

Participants continue to receive DYNE-101 every 8 weeks for up to 24 weeks

24 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• zeleciment basivarsen (DYNE-101)

How Is the Trial Designed?

Group I: Placebo-Controlled Period: DYNE-101 Experimental Treatment 1 Intervention

Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.

Group II: Long-Term Extension Period: DYNE-101 Experimental Treatment 2 Interventions

Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.

Group III: Placebo-Controlled Period: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.

Who Is Running the Clinical Trial?

Dyne Therapeutics

Lead Sponsor

Trials

3

Recruited

640+