NMD670 for Spinal Muscular Atrophy

(SYNAPSE-SMA Trial)

Recruiting at 29 trial locations

Overseen By NMD Pharma A/S

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: NMD Pharma A/S

Disqualifiers: Scoliosis, Neuromuscular diseases, others

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

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 3 weeks

54 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you have taken an investigational medical product recently, you may need to wait 30 days or longer before joining the trial.

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

Eligibility Criteria

Adults with Type 3 Spinal Muscular Atrophy (SMA) who can walk at least 50 meters unaided, have genetic confirmation of SMA, and a BMI of 19-30 kg/m2. They must use contraception and be able to consent to the study. Excluded are those with recent investigational drug use, surgeries or deformities affecting movement, other significant diseases, or poor compliance with SMA therapy.

Inclusion Criteria

Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

I have been diagnosed with Type 3 Spinal Muscular Atrophy.

I can walk more than 50 metres without help.

Exclusion Criteria

I do not have any major diseases that could affect the study's results.

I have not followed my SMA treatment plan as recommended.

Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either the experimental drug or placebo in a 2-way crossover design

3 weeks

Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• NMD670

Trial Overview The trial is testing NMD670's effectiveness and safety for adults with Type 3 SMA compared to a placebo. It will also look at how the body processes the drug. Participants will randomly receive either NMD670 or a placebo in this controlled study.

Participant Groups

Group I: Cohort 2 Experimental Treatment 2 Interventions

Placebo followed by experimental drug

Group II: Cohort 1 Experimental Treatment 2 Interventions

Experimental drug followed by placebo

NMD670 is already approved in United States for the following indications:

Approved in United States as NMD670 for:

• Generalized Myasthenia Gravis (gMG)
• Charcot-Marie-Tooth disease (CMT)

Who Is Running the Clinical Trial?

NMD Pharma A/S

Lead Sponsor

Trials

6

Recruited

290+