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Small Molecule

NMD670 for Spinal Muscular Atrophy (SYNAPSE-SMA Trial)

Phase 2
Recruiting
Research Sponsored by NMD Pharma A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participants with a clinical diagnosis of Type 3 SMA
Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids
Must not have
Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to day 21

Summary

This trial tests if NMD670 is a safe and effective treatment for adults with spinal muscular atrophy type 3.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects for NMD670 aren't listed here, common ones may include reactions at the injection site, fatigue, gastrointestinal issues like nausea or diarrhea, muscle pain, and potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with Type 3 Spinal Muscular Atrophy.
Select...
I can walk more than 50 metres without help.
Select...
My condition was confirmed through genetic testing to be due to a specific gene deletion.
Select...
I have 3 to 5 copies of the SMN2 gene.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I don't have any physical conditions like scoliosis that would stop me from doing study tasks.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to day 21
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to day 21 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Secondary study objectives
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Change from baseline in blocking versus placebo
+10 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2Experimental Treatment2 Interventions
Placebo followed by experimental drug
Group II: Cohort 1Experimental Treatment2 Interventions
Experimental drug followed by placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

NMD Pharma A/SLead Sponsor
5 Previous Clinical Trials
237 Total Patients Enrolled

Media Library

NMD670 (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT05794139 — Phase 2
Spinal Muscular Atrophy Research Study Groups: Cohort 1, Cohort 2
Spinal Muscular Atrophy Clinical Trial 2023: NMD670 Highlights & Side Effects. Trial Name: NCT05794139 — Phase 2
NMD670 (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05794139 — Phase 2
~18 spots leftby Jul 2025
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