NMD670 for Spinal Muscular Atrophy

(SYNAPSE-SMA Trial)

Recruiting at 29 trial locations

Overseen By NMD Pharma A/S

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: NMD Pharma A/S

Disqualifiers: Scoliosis, Neuromuscular diseases, others

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

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

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.

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

Trial 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 (Small Molecule)

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

Who Is Running the Clinical Trial?

NMD Pharma A/S

Lead Sponsor

Trials

6

Recruited

290+