NMD670 for Spinal Muscular Atrophy

(SYNAPSE-SMA Trial)

Recruiting at 34 trial locations

Overseen By NMD Pharma A/S

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: NMD Pharma A/S

Disqualifiers: Scoliosis, Neuromuscular diseases, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 3 weeks

54 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called NMD670 (Bromophenoxyazole propanoic acid) for individuals with spinal muscular atrophy type 3 (SMA3) to determine its potential benefits. The study will assess the drug's safety, effectiveness, and tolerability. Participants will be divided into two groups: one will receive the drug first, followed by a placebo (a pill with no active ingredient), and the other will receive them in reverse order. The trial seeks ambulatory adults with SMA3 who can walk at least 50 meters unassisted. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that NMD670 is likely to be safe for humans?

Research has shown that NMD670 produced positive results in earlier studies with patients who have myasthenia gravis, a long-term disease affecting the muscles. These studies suggest that the treatment is generally safe and did not cause major side effects. Now in Phase 2 trials, NMD670 has already passed initial safety tests in humans, increasing confidence in its safety for participants. While the current study focuses on spinal muscular atrophy type 3, the earlier findings help reassure about its overall safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for spinal muscular atrophy?

Most treatments for Spinal Muscular Atrophy (SMA), like nusinersen and risdiplam, work by boosting the production of a protein critical for motor neuron survival. But NMD670 works differently, targeting the underlying cause of SMA with a novel mechanism of action. Researchers are excited about NMD670 because it aims to directly enhance muscle function, potentially leading to improvements in strength and mobility beyond what existing treatments offer. This unique approach could fill a crucial gap for patients who need more than just increased protein production.

What evidence suggests that NMD670 might be an effective treatment for spinal muscular atrophy?

Research has shown that NMD670 has produced promising results in early studies for conditions like myasthenia gravis, which involve problems at the connection between nerves and muscles. This is significant because similar issues occur in spinal muscular atrophy (SMA). NMD670 blocks a specific channel in cells, potentially improving muscle function. Although specific data for SMA type 3 is limited, the mechanism of NMD670 suggests it could enhance muscle strength and function in individuals with this condition. Early signs from related studies support the possibility of positive outcomes for those with SMA type 3. Participants in this trial will receive either the experimental drug followed by a placebo or a placebo followed by the experimental drug, enabling researchers to evaluate the potential benefits of NMD670 for SMA. ^{1 2 3 5 6}

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

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• NMD670

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.

How Is the Trial Designed?

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

NMD670 is already approved in United States for the following indications:

Approved in United States as NMD670 for:

• Generalized Myasthenia Gravis (gMG)
• Charcot-Marie-Tooth disease (CMT)

Who Is Running the Clinical Trial?

NMD Pharma A/S

Lead Sponsor

Trials

6

Recruited

290+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05794139?utm_source=trialradar

Safety and Efficacy of NMD670 in Ambulatory Adult ... The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal ...

2. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/430396

Safety and Efficacy of NMD670 in Ambulatory Adult ... The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory ...

3. nmdpharma.com nmdpharma.com/news/nmd-initiates-phase-2-trial-of-nmd670-in-sma

NMD Pharma initiates Phase II trial of NMD670 in Spinal ... The first patient has been dosed in a Phase II clinical trial with the ClC-1 inhibitor NMD670 in patients impacted by spinal muscular atrophy (SMA).

4. nmdpharma.com nmdpharma.com/news/nmd-pharma-initiates-phase-2-study-of-nmd670-in-patients-with-cmt-type-1-and-2

NMD Pharma Initiates Phase 2 Study of NMD670 in ... NMD670 has demonstrated promising clinical results in a proof-of-mechanism study in patients with NMJ dysfunction in the rare neuromuscular disease myasthenia ...

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05794139/safety-and-efficacy-of-nmd670-in-ambulatory-adult-patients-with-type-3-spinal-muscular-atrophy

Safety and Efficacy of NMD670 in Ambulatory Adult ... The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory ...

6. uclahealth.org uclahealth.org/clinical-trials/safety-and-efficacy-nmd670-ambulatory-adult-patients-with

Safety and Efficacy of NMD670 in Ambulatory Adult ... Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy. About. Brief Summary. The purpose of this study is to evaluate ...