Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)
Spinal Muscular Atrophy
About this trial
This is an interventional treatment trial for Spinal Muscular Atrophy focused on measuring Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1
Eligibility Criteria
Inclusion Criteria: 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. Participant with genetic confirmation of diagnosis (i.e., homozygous deletion 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 19-30 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. Exclusion Criteria: 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.
Sites / Locations
- UF Fixel Institute for Neurological DiseasesRecruiting
- University of Kansas Medical CenterRecruiting
- Roy Blunt NextGen Precision Health InstituteRecruiting
- Washington University School of MedicineRecruiting
- The Ohio State University Wexner Medical CenterRecruiting
- UZ Leuven - Neurochirurgie Campus GasthuisbergRecruiting
- CHR de la Citadelle - NeurologieRecruiting
- Aarhus Universitetshospital, Neurologisk AfdelingRecruiting
- Rigshospitalet - Neurologisk AfdelingRecruiting
- Charite - Campus Virchow-Klinikum (CVK)Recruiting
- Universitätsklinikum Essen - Klinik Für NeurologieRecruiting
- Istituto Neurologico C. Besta, Fondazione IRCCSRecruiting
- Ospedale Niguarda, ASST Grande Ospedale Metropolitano NiguardaRecruiting
- Universitair Medisch Centrum Utrecht, locatie Academisch Zie - NeurologyRecruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Cohort 1
Cohort 2
Experimental drug followed by placebo
Placebo followed by experimental drug