Spinal Muscular AtrophyPhase 2SYNAPSE-SMANCT05794139
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
A Phase 2 study testing NMD670 in people with Spinal Muscular Atrophy, run by NMD Pharma A/S at 25 sites in 8 countries.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- NMD670
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 25 sitesin 8 countries
- Study dates
- 21 Sep 2023to 18 May 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 at screening during the 6-minute walk test.
- Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])
- Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
- Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
- Participant is male or female.
- Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Participant is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
Who cannot join
- Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
- Participants received treatment with an investigational medical product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1.
- Participants with history of poor compliance with relevant SMA therapy.
Trial locations and contacts
25 sites in 8 countries.
25 sites in 8 countries
United States9 sites
The Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore, Maryland, 21287
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, 65212
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Neurology Rare Disease Center
Denton, Texas, 76208
Rare Disease Research - Raleigh-Durham
Hillsborough, North Carolina, 27278
University of Kansas Medical Center
Kansas City, Kansas, 66160
UCLA David Geffen School Of Medicine - Neurology
Los Angeles, California, 90095
Stanford University Medical Center
Palo Alto, California, 94304
Washington University School of Medicine
St Louis, Missouri, 63110
Italy4 sites
Istituto Giannina Gaslini, IRCCS
Genova
Istituto Neurologico C. Besta, Fondazione IRCCS
Milan
Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
Milan
AOU Città della Salute e della Scienza di Torino
Torino
Spain3 sites
Hospital Universitari Vall D Hebron
Barcelona
Hospital Materno Infantil La Paz
Madrid
Hospital Universitario y Politécnico La Fe
Valencia
Belgium2 sites
UZ Leuven - Neurochirurgie Campus Gasthuisberg
Leuven
CHR de la Citadelle - Neurologie
Liège
Canada2 sites
Heritage Medical Research Clinic
Calgary
Genge Partners Inc.
Montreal
Denmark2 sites
Aarhus Universitetshospital, Neurologisk Afdeling
Aarhus
Rigshospitalet - Neurologisk Afdeling
Copenhagen
Germany2 sites
Charite - Campus Virchow-Klinikum (CVK)
Berlin
Universitätsklinikum Essen - Klinik Für Neurologie
Essen
Netherlands1 site
Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology
Utrecht
About this study
Official title: A Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy
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
- Conditions
- Spinal Muscular Atrophy
- Being tested
- NMD670, Placebo
- Keywords
- Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NMD670Drug
Tablets
PlaceboDrug
Tablets
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Experimental drug followed by placebo Receives: Drug: NMD670; Drug: Placebo |
| Cohort 2 | Experimental | Placebo followed by experimental drug Receives: Drug: NMD670; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Distance walked (meters)
Measured: Baseline to day 21
Secondary outcomes (13)
Change from baseline in muscle strength versus placebo
Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)
Measured: Baseline to day 21
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
percentage change in distance walked in 6th minute compared to 1st minute
Measured: Baseline to day 21
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms
Measured: Baseline to day 21
Change from baseline in jitter versus placebo
Jitter (micro seconds) assessed with single fiber EMG
Measured: Baseline to day 21
Change from baseline in blocking versus placebo
Blocking (%) assessed with single fiber EMG
Measured: Baseline to day 21
Incidence of treatment emergent adverse events
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of serious treatment emergent adverse events
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant ECG abnormalities
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations
Summarised per treatment
Measured: Over 21 days of dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2023
- Study start21 Sep 2023
- Primary completion11 May 2026
- Study completion18 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 2-way crossover
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- NMD Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05794139
- Sponsor's protocol ID
- NMD670-02-0001
- Other IDs
- 2022-002301-24
- Acronym
- SYNAPSE-SMA
- Status last verified
- Jun 2026
- First submitted
- 15 Mar 2023
- Last updated
- 3 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.
PubMed 41224659
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT05794139
- Acronym / sponsor ID
- SYNAPSE-SMA / NMD670-02-0001
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- NMD Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- 21 Sep 2023 (actual)
- Primary completion
- 11 May 2026 (actual)
- Study completion
- 18 May 2026 (actual)
- First posted
- 3 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 2-way crossover
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: NMD670, Drug: PlaceboExperimental drug followed by placebo |
| Cohort 2 | Experimental | Drug: NMD670, Drug: PlaceboPlacebo followed by experimental drug |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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 at screening during the 6-minute walk test.
- Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])
- Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
- Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
Key exclusion criteria (5 listed)
- Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
- Participants received treatment with an investigational medical product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1.
- Participants with history of poor compliance with relevant SMA therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NMD670Tablets | Drug | Cohort 1, Cohort 2 |
| PlaceboTablets | Drug | Cohort 1, Cohort 2 |
Primary outcome
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Time frame: Baseline to day 21
Distance walked (meters)
13 secondary outcomes
Change from baseline in muscle strength versus placebo
Time frame: Baseline to day 21
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
Time frame: Baseline to day 21
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Time frame: Baseline to day 21
Change from baseline in jitter versus placebo
Time frame: Baseline to day 21
Change from baseline in blocking versus placebo
Time frame: Baseline to day 21
Incidence of treatment emergent adverse events
Time frame: Over 21 days of dosing
Incidence of serious treatment emergent adverse events
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters
Time frame: Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities
Time frame: Over 21 days of dosing
Incidence of clinically significant ECG abnormalities
Time frame: Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations
Time frame: Over 21 days of dosing
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Derived from this study: PubMed 41224659Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
25 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NMD670 in people with Spinal Muscular Atrophy, run by NMD Pharma A/S at 25 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Italy, Spain, Belgium, Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo (time frame: Baseline to day 21). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05794139, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2026.