NMD670 for Spinal Muscular Atrophy
(SYNAPSE-SMA Trial)
What You Need to Know Before You Apply
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.
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either the experimental drug or placebo in a 2-way crossover design
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- NMD670
NMD670 is already approved in United States for the following indications:
- Generalized Myasthenia Gravis (gMG)
- Charcot-Marie-Tooth disease (CMT)
Find a Clinic Near You
Who Is Running the Clinical Trial?
NMD Pharma A/S
Lead Sponsor