Study
Spinal muscular atrophy (SMA) is a rare, genetic condition affecting nerves that carry impulses that act as messages from the brain to control skeletal muscle (muscles in hands, legs, chest, face, throat, etc.) and lead to muscle activation and movement. In people with SMA, many of the motor nerves have reduced levels of a protein called SMN that leads to muscle weakness and fatigue, affecting mobility and independence. Even with modern treatments meant to maintain or increase SMN protein for the motor nerves, many still experience disabling symptoms. That’s why researchers are now exploring new approaches, such as medicines designed to directly improve how muscles respond and function, to help people living with SMA regain strength and stamina in daily life.
What Is Spinal Muscular Atrophy?
About NMD670
NMD670 is an investigational oral medicine (drug) being studied as a possible treatment for people with SMA.
It works differently from other SMA treatments by helping muscles respond to nerve signals. Early research suggests it may help muscles activate more effectively, even when signals from the nerves are weak. This could lead to improvements in muscle strength and reduce muscle fatigue The purpose of this clinical trial is to learn how well different doses of the study drug work and how safe it is compared to a placebo.
The study drug is taken as 2 tablets twice a day with water and can be used in addition to your current SMA medications.
In an earlier clinical trial with a small group of people living with myasthenia gravis (a different rare neuromuscular disease), patients received only a single dose of NMD670 and results demonstrated meaningful improvements in muscle strength for patients across multiple types of skeletal muscles (face, mouth/swallowing, arms, legs) throughout the body. Now, NMD Pharma and clinical researchers across a number of countries are testing NMD670 in this larger clinical trial in SMA to understand the safety and efficacy of the drug in various dosing levels over 21 days.
SYNAPSE-SMA Clinical Trial
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Find a convenient location
Look for a study site that works best for you.
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Do you qualify for the study?
Answer a few simple questions to find out if you're eligible.
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We'll be in touch
If you're eligible, someone from our team will reach out to answer your questions and help you schedule an appointment.
I felt empowerment in my ability to participate in a clinical trial that might one day help myself, my son and many others to come.
Clinical Trial ParticipantAbout the SMA Clinical Trial
The purpose of this clinical trial is to study the safety and effects of NMD670 in adults with SMA.
- Taking 2 tablets twice a day with water for 42 days
- Participate in 6 in-person study visits over about 13 weeks and 4 phone check-ins
- Agreement to take muscle strength assessments, walking tests, blood tests, electrocardiograms (ECGs), and to complete questionnaires as well as a diary of time and date when study drug was taken
- All study participants will receive NMD670 at some point during the study, but also placebo at some point
- Participants may continue taking their current SMA treatment
- Travel costs will be reimbursed, with possible compensation for time or lost wages
- Participation could include a stipend of up to $300/per day for each day that a patient attends an in-clinic visit
- Participation is voluntary and at no cost to the participant
- Ambulatory adults aged 18–75
- Those with a confirmed diagnosis of Type 3 SMA
- Able to walk at least 164 feet without walking aids
- No prior surgery or a condition (such as scoliosis or contractures) that restricts your abilities
- No other significant disease (such as other neuromuscular or muscular diseases)
See if You Qualify
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