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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to day 21

Awards & highlights

SYNAPSE-SMA Trial 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.Check my eligibility

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.See study design

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.

SYNAPSE-SMA Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Type 3 Spinal Muscular Atrophy.

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I can walk more than 50 metres without help.

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My condition was confirmed through genetic testing to be due to a specific gene deletion.

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I have 3 to 5 copies of the SMN2 gene.

SYNAPSE-SMA Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to day 21

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in 6 minute walk test (6MWT) total distance versus placebo

Secondary outcome measures

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

SYNAPSE-SMA 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

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

NMD Pharma A/SLead Sponsor

3 Previous Clinical Trials

73 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the participant pool of this experiment composed of individuals over 75 years old?

"The age range for involvement in this trial is listed as 18-60, so any participants younger or older than that are ineligible."

Answered by AI

Has Cohort 2 completed the necessary regulatory requirements to receive FDA authorization?

"Cohort 2's safety was judged to be a level 2 on the scale due to limited data supporting its efficacy, though there is some evidence indicating it may still be safe."

Answered by AI

To what degree is patient participation in this trial substantial?

"NMD Pharma A/S, the sponsor of this clinical trial, is recruiting 54 patients that meet its criteria to participate in the study. The research will be conducted at University of Kansas Medical Center and Roy Blunt NextGen Precision Health Institute respectively located in Kansas City and Columbia."

Answered by AI

How many settings are providing access to this research?

"Currently, 4 different sites are involved in this trial. Kansas City, Columbia and Copenhagen are the main cities; however, there is also additional locations nearby. It's recommended to find a centre closest to your home for ease of access during participation."

Answered by AI

What qualifications are necessary to participate in this clinical research?

"This trial is recruiting 54 participants aged 18 to 60 with spinal muscular atrophy. In addition, they must have a clinical diagnosis of type 3 SMA; the ability to walk more than 50 metres without aid; genetic confirmation of their condition; 3-5 copies of SMN2 gene; BMI between 19 and 30 kg/m2 (inclusive); be male or female and capable of giving informed consent that includes understanding all requirements and restrictions."

Answered by AI

Are there any openings for participants in this trial currently?

"According to clinicaltrials.gov, this trial is currently recruiting participants. The initial posting was on August 1st 2023 and the most recent changes were made July 14th of that same year."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

2023. "Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05794139.

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