DYNE-101 for Myotonic Dystrophy

(ACHIEVE Trial)

Recruiting at 15 trial locations

Overseen By Dyne Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Dyne Therapeutics

Must not be taking: Myotonia medications, GLP-1 agonists

Disqualifiers: Major surgery, Anaphylaxis, ECG abnormalities, others

Compensation: Varies

Number of Visits: Unknown

116 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and tolerability of the new treatment, DYNE-101, for individuals with Myotonic Dystrophy Type 1 (DM1), a condition causing muscle weakness and stiffness. Participants will receive DYNE-101 through intravenous infusion, with various dosing schedules to determine the most effective approach. The trial includes different phases, such as a placebo-controlled part and a long-term extension to observe effects over time. It suits those who have experienced DM1 muscle symptoms since at least age 12, have noticeable muscle stiffness (myotonia), and can complete certain physical tasks like walking and climbing stairs without assistance. As a Phase 1 and Phase 2 trial, this study aims to understand how DYNE-101 works in people and measure its effectiveness in an initial group, offering participants a chance to contribute to groundbreaking research.

Do I need to stop my current medications to join the trial?

You may need to stop taking certain medications that improve myotonia or GLP-1 agonist medications before joining the trial. The protocol mentions a period of 5 half-lives (time it takes for the drug's active substance to reduce by half in the body) for these medications before screening assessments.

Is there any evidence suggesting that DYNE-101 is likely to be safe for humans?

Research has shown that DYNE-101 is generally safe. In studies, most side effects appeared mild or moderate and are known as treatment-emergent adverse events (TEAEs). Importantly, researchers found no serious side effects linked to the treatment. This suggests that patients usually tolerate DYNE-101 well. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about DYNE-101 because it represents a novel approach to treating myotonic dystrophy. Unlike traditional treatments that primarily focus on managing symptoms, DYNE-101 aims to target the underlying genetic causes of the disease. This treatment utilizes a unique mechanism that delivers therapeutic agents directly to muscle cells, potentially addressing the root cause rather than just alleviating symptoms. By doing so, DYNE-101 has the potential to offer more long-lasting benefits and improve overall muscle function, which sets it apart from existing therapies.

What evidence suggests that DYNE-101 might be an effective treatment for Myotonic Dystrophy?

Research has shown that DYNE-101, which participants in this trial may receive, offers promising results for treating Myotonic Dystrophy Type 1 (DM1). One study demonstrated that patients receiving a 1.8 mg/kg dose every four weeks improved by 39% after 12 months. Another study found positive trends with a 6.8 mg/kg dose every eight weeks, showing improvements in how patients felt and functioned. These findings suggest DYNE-101 may significantly help people with DM1 by addressing the root causes of the disease. While more research is needed, the early results are encouraging for those considering this treatment. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for people with Myotonic Dystrophy Type 1 (DM1) who have a specific genetic marker, started experiencing muscle symptoms at age 12 or older, and can perform certain physical tests without help. They should also have a moderate level of hand grip and ankle strength.

Inclusion Criteria

Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator

I have DM1 with a genetic marker over 100 repeats.

My hand grip and ankle strength are normal.

Exclusion Criteria

History of anaphylaxis

My lung function is severely reduced.

I haven't taken myotonia medication 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 receive either DYNE-101 or placebo in a randomized manner

24 weeks

Treatment Period

Participants continue to receive DYNE-101, with those initially on placebo switching to active treatment

24 weeks

Long-Term Extension (LTE) Period

Participants receive DYNE-101 for an extended period to assess long-term effects

168 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• DYNE-101
• Placebo

Trial Overview The study is testing DYNE-101, given through an IV, to see if it's safe and tolerable for DM1 patients. It includes a placebo-controlled period followed by treatment and long-term extension phases over several years.

How Is the Trial Designed?

Group I: MAD Cohort: Treatment Period: DYNE-101 Experimental Treatment 2 Interventions

Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.

Group II: MAD Cohort: Placebo-Controlled Period: DYNE-101 Experimental Treatment 1 Intervention

Participants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks.

Group III: MAD Cohort: Long-Term Extension Period: DYNE-101 Experimental Treatment 1 Intervention

Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.

Group IV: Dose Expansion Cohort: Treatment Period: DYNE-101 Experimental Treatment 2 Interventions

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

Group V: Dose Expansion Cohort: Placebo-Controlled Period: Placebo Experimental Treatment 1 Intervention

Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.

Group VI: Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101 Experimental Treatment 1 Intervention

Participants will receive DYNE-101, Q8W for up to 24 weeks.

Group VII: Dose Expansion Cohort: Long-Term Extension Period: DYNE-101 Experimental Treatment 1 Intervention

Participants will receive DYNE-101, Q8W for up to 168 weeks.

Group VIII: MAD Cohort: Placebo-Controlled Period: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.

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-additional-one-year-clinical-data

Dyne Therapeutics Announces Additional One-Year ... ... Outcomes: Meaningful and sustained improvement from baseline in the Myotonic Dystrophy ... 101) in patients with myotonic dystrophy type 1 (DM1).

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000210508

Safety and Efficacy of DYNE-101 in Adults with DM1 In the 1.8 mg/kg Q4W cohort, DYNE-101 resulted in continued improvement in vHOT, from a 28% change from baseline at 3 months to 39% change at 12 ...

3. mdaconference.org mdaconference.org/abstract-library/safety-and-efficacy-of-dyne-101-in-adults-with-dm1-phase-1-2-achieve-trial-data/

Safety and Efficacy of DYNE-101 in Adults with DM1 In the 1.8 mg/kg Q4W cohort, DYNE-101 resulted in continued improvement in vHOT, from 28% change from baseline at 3 months to 39% change at 12 months. The 5.4 ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05481879

Study Details | NCT05481879 | Safety, Tolerability, ... The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with ...

5. investors.dyne-tx.com investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-reports-new-clinical-data-showing-compelling

Dyne Plans to Initiate Registrational Expansion Cohort ... Myotonic Dystrophy Health Index (MDHI): DYNE-101 at the 6.8 mg/kg Q8W doses showed encouraging trends on the MDHI patient reported outcome (PRO) ...

6. investors.dyne-tx.com investors.dyne-tx.com/static-files/e05f7874-d149-4f83-8dcc-b0bb686f9aa7

DYNE-101 US Accelerated Approval and Clinical Update Safety data as of April 23, 2025; DM1 = myotonic dystrophy type 1; MAD = multiple ascending dose; LTE = long term extension; CASI-22 ...