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Safety and efficacy of NMD670 in ambulatory adult patients with Type 3 spinal muscular atrophy (SYNAPSE-SMA)
EU CTIS ID: 2024-516321-31-00
What this study is testing
To assess changes in muscle strength and function after NMD670 400 mg bid for 21 days, compared with placebo, in participants with SMA
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participant must be 18 to 75 years of age inclusive, at the time of signing the informed consent.
- Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male Participants: - A male participant must agree to use a highly effective contraception as detailed in Appendix 4 of this protocol during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention) and refrain from donating sperm during this period. Female Participants: - A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies: - Not a woman of childbearing potential (WOCBP) as defined in Appendix 4. OR - A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention).
- Participant is capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Participants who are 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]).
You likely can't join if
- Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- Participants unable to perform or tolerate electromyography (EMG), according to medical history or at screening.
- Participants with history of poor compliance with relevant SMA therapy.
- Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- Participant with a clinical diagnosis of gout, or with serum uric acid >ULN at screening.
- Participant with clinically significant electrocardiogram (ECG) abnormalities at screening including PR interval ≥220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare supraventricular or rare ventricular ectopic beats) in the judgement of the Investigator, or T-wave configurations are not of sufficient quality for assessing QT interval duration.
See the full eligibility criteria
Who can join
- Participant must be 18 to 75 years of age inclusive, at the time of signing the informed consent.
- Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male Participants: - A male participant must agree to use a highly effective contraception as detailed in Appendix 4 of this protocol during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention) and refrain from donating sperm during this period. Female Participants: - A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies: - Not a woman of childbearing potential (WOCBP) as defined in Appendix 4. OR - A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention).
- Participant is capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Participants who are 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 2 to 5 copies of survival of motor neuron 2 gene [SMN2].
- Participants with an MFM-32 dimension 1 (D1) score <80% at screening.
- Participants with ≥7% CMAP amplitude decrement at screening during repeated nerve stimulation [RNS].
- Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
- Participant is male or female.
What rules you out
- Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- Participants unable to perform or tolerate electromyography (EMG), according to medical history or at screening.
- Participants with history of poor compliance with relevant SMA therapy.
- Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- Participant with a clinical diagnosis of gout, or with serum uric acid >ULN at screening.
- Participant with clinically significant electrocardiogram (ECG) abnormalities at screening including PR interval ≥220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare supraventricular or rare ventricular ectopic beats) in the judgement of the Investigator, or T-wave configurations are not of sufficient quality for assessing QT interval duration.
- Participant with any of the following abnormalities in QT interval corrected for heart rate using Fridericia’s correction (QTcF) at screening: a.QTcF interval greater than 450 msec at baseline for males and 460 msec for females; b. PR interval greater than 220 msec; c. complete bundle branch block (QRS ≥120 msec)
- Participant with any of the following: a. Abnormal liver function test defined as total bilirubin >1.5×ULN (participants with Gilbert's syndrome can be included with total bilirubin >1.5×ULN if direct bilirubin is ≤1.5×ULN and ≤35% of total bilirubin). b. Abnormal liver transaminase levels at baseline or current or chronic history of liver disease including (but not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non-alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson's disease, α-1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease considered clinically significant by the Investigator. c. Known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones). d. Abnormal renal function with estimated glomerular filtration rate (eGFR), calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation based on cystatin C: < 40 years: eGFR < 75 mL/min/1.73 m². 40-65 years: eGFR < 60 mL/min/1.73 m². >65 years: eGFR < 45 mL/min/1.73 m², provided there is no proteinuria.
- Participant with clinically significant laboratory test abnormalities at screening.
- Participant with breast cancer within the past 10 years or lymphoma, leukaemia, or any malignancy within the past 5 years. An exception of this 5-year requirement is basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease.
- Participants with ongoing significant psychiatric disorder (e.g., uncontrolled depression, anxiety).
- Participants with positive drug screen (cocaine, heroin, opiates, and ketamine) at screening. Participants will not be excluded from the study if they showed a positive drug screen due to medically prescribed opiates or ketamine.
- Participants with Myotonic disorders or those on drugs that induce or mask myotonia.
- Participants with positive human immunodeficiency virus (HIV) antibody test.
- Participants with presence of hepatitis B surface antigen (HBsAg) [or hepatitis B core antibody (HBcAb)] at screening or within 3 months prior to first dose of investigational intervention.
- Participants with positive hepatitis C antibody or ribonucleic acid (RNA) test result at screening or within 3 months prior to Day 1. NOTE: Participants with hepatitis C with positive hepatitis C antibody could be enrolled, if a negative hepatitis C RNA test is obtained.
- Participants with a clinically significant history of allergic conditions (including drug allergies and anaphylactic reactions) or hypersensitivity to any component of the study intervention.
- Participants unable to undergo the ophthalmologic evaluation, at screening.
- Participants who have received any prohibited medication within 5 half-lives of the medication, prior to day 1 or are likely to require treatment with prohibited medications during the study. Prohibited medications are listed in Section 6.9 and include drugs affecting neuromuscular transmission (such as anticholinergic drugs), drugs showing relevant effect on ClC-1 channel, drugs with potential drug-drug interactions with NMD670. Other current and recent (within 1 month prior to the screening) treatments will be allowed, if judged by the Investigator to have no clinical relevance.
- Participants received treatment with an investigational medicinal product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.