- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05794139
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)
April 24, 2024 updated by: NMD Pharma A/S
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
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
54
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: NMD Pharma A/S
- Phone Number: contact@nmdpharma.com
- Email: contact@nmdpharma.com
Study Locations
-
-
-
Leuven, Belgium
- Recruiting
- UZ Leuven - Neurochirurgie Campus Gasthuisberg
-
Contact:
- Joyce Cremers
- Phone Number: 0032-16347361
- Email: joyce.cremers@uzleuven.be
-
Contact:
- Elisa Debien
- Phone Number: 0032-16341743
- Email: elisa.debien@uzleuven.be
-
Liège, Belgium
- Recruiting
- CHR de la Citadelle - Neurologie
-
-
-
-
-
Calgary, Canada
- Recruiting
- Heritage Medical Research Clinic
-
Contact:
- Berchman Wong
- Email: wongb@ucalgary.ca
-
Montréal, Canada
- Recruiting
- Genge Partners Inc.
-
Contact:
- Vincent Etten
- Email: vetten@gengepartners.com
-
-
-
-
-
Aarhus, Denmark
- Recruiting
- Aarhus Universitetshospital, Neurologisk Afdeling
-
Contact:
- Josefine Nielsen
- Email: JOSENL@rm.dk
-
København, Denmark
- Recruiting
- Rigshospitalet - Neurologisk Afdeling
-
Contact:
- Nicolai Preisler
- Email: nicolai.rasmus.preisler@regionh.dk
-
-
-
-
-
Berlin, Germany
- Recruiting
- Charite - Campus Virchow-Klinikum (CVK)
-
Essen, Germany
- Recruiting
- Universitätsklinikum Essen - Klinik Für Neurologie
-
Contact:
- Jaqueline Lipka
- Phone Number: +49201-72385563
- Email: Jaqueline.lipka@uk-essen.de
-
Contact:
- Benjamin Stolte
- Email: Benjamin.stolte@uk-essen.de
-
-
-
-
-
Genova, Italy
- Recruiting
- Istituto Giannina Gaslini, IRCCS
-
Milano, Italy
- Recruiting
- Istituto Neurologico C. Besta, Fondazione IRCCS
-
Milano, Italy
- Recruiting
- Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
-
Pisa, Italy
- Recruiting
- Università degli studi di Pisa
-
Contact:
- Giulia Ricci
- Phone Number: +39 3337013195
- Email: giulia.ricci2304@gmail.com
-
Roma, Italy
- Recruiting
- PU A. Gemelli, Università Cattolica del Sacro Cuore
-
Contact:
- Eugenio Mercuri
- Phone Number: +39 0630158576
- Email: eugeniomaria.mercuri@policlinicogemelli.it
-
-
-
-
-
Utrecht, Netherlands
- Recruiting
- Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology
-
-
-
-
-
Barcelona, Spain
- Recruiting
- Hospital Universitari Vall d Hebron
-
Contact:
- Javier Sotoca
- Phone Number: 2780 +34934893000
- Email: Sotocajavier.sotoca@vallhebron.cat
-
Donostia, Spain
- Recruiting
- H. Donostia
-
Contact:
- Ioana Croitoru
- Phone Number: +34943007027
- Email: IOANA.CROITORU@bio-gipuzkoa.eus
-
Madrid, Spain
- Recruiting
- Hospital Materno Infantil La Paz
-
Contact:
- María Romero
- Phone Number: +34917277388
- Email: mmar.garcia.romero@salud.madrid.org
-
Valencia, Spain
- Recruiting
- Hospital Universitario y Politécnico La Fe
-
Contact:
- Paula Cortés
- Phone Number: 411748 +34961244000
- Email: ame_lafe@gva.es
-
-
-
-
California
-
Los Angeles, California, United States, 90095
- Recruiting
- UCLA David Geffen School Of Medicine - Neurology
-
Palo Alto, California, United States, 94304
- Recruiting
- Stanford University Medical Center
-
Contact:
- Lidia Choniawko
- Phone Number: 650-304-8094
- Email: lidiacho@stanford.edu
-
-
Florida
-
Gainesville, Florida, United States, 32608
- Recruiting
- UF Fixel Institute for Neurological Diseases
-
Contact:
- Beverly Brooks
- Phone Number: 352-733-2425
- Email: Beverly.brooks@neurology.ufl.edu
-
-
Georgia
-
Atlanta, Georgia, United States, 30329
- Recruiting
- Rare Disease Center
-
Contact:
- Darrell Favors
- Email: darrell.favors@rarediseaseresearch.com
-
-
Kansas
-
Kansas City, Kansas, United States, 66160
- Recruiting
- University of Kansas Medical Center
-
Contact:
- Cassidy Nelson
- Phone Number: 913-588-3653
- Email: cnelson15@kumc.edu
-
-
Missouri
-
Columbia, Missouri, United States, 65212
- Recruiting
- Roy Blunt NextGen Precision Health Institute
-
Contact:
- Neetha Gali
- Phone Number: 573-882-3065
- Email: ngdcd@missouri.edu
-
Saint Louis, Missouri, United States, 63110
- Recruiting
- Washington University School of Medicine
-
Contact:
- Jessie Schwieterman
- Phone Number: 314-362-1566
- Email: j.schwieterman@wustl.edu|phone:
-
-
North Carolina
-
Hillsborough, North Carolina, United States, 27278
- Recruiting
- Rare Disease Research - Raleigh-Durham
-
Contact:
- Annette Babu
- Phone Number: 984-314-2252
- Email: annette.babu@rarediseaseresearch.com
-
-
Ohio
-
Columbus, Ohio, United States, 43210
- Recruiting
- The Ohio State University Wexner Medical Center
-
Contact:
- Gia Cinkay
- Phone Number: 614-366-9050
- Email: georgia.cinkay@osumc.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 60 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- 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.
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1
Experimental drug followed by placebo
|
Tablets
Tablets
|
Experimental: Cohort 2
Placebo followed by experimental drug
|
Tablets
Tablets
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Time Frame: Baseline to day 21
|
Distance walked (meters)
|
Baseline to day 21
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in muscle strength versus placebo
Time Frame: Baseline to day 21
|
Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)
|
Baseline to day 21
|
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
Time Frame: Baseline to day 21
|
percentage change in distance walked in 6th minute compared to 1st minute
|
Baseline to day 21
|
Change from baseline in jitter versus placebo
Time Frame: Baseline to day 21
|
Jitter (micro seconds) assessed with single fiber EMG
|
Baseline to day 21
|
Change from baseline in blocking versus placebo
Time Frame: Baseline to day 21
|
Blocking (%) assessed with single fiber EMG
|
Baseline to day 21
|
Incidence of treatment emergent adverse events
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of serious treatment emergent adverse events
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of clinically significant abnormalities on physical examinations
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of clinically significant abnormalities on safety laboratory parameters
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of clinically significant vital signs abnormalities
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of clinically significant ECG abnormalities
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Time Frame: Baseline to day 21
|
Total score.
Scale goes from 0-69 and higher score indicates improvement of symptoms
|
Baseline to day 21
|
Incidence of Suicidal Ideation or Suicidal Behavior
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Incidence of clinically significant abnormalities on opthalmological examinations
Time Frame: Over 21 days of dosing
|
Summarised per treatment
|
Over 21 days of dosing
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 21, 2023
Primary Completion (Estimated)
December 1, 2024
Study Completion (Estimated)
December 1, 2024
Study Registration Dates
First Submitted
March 15, 2023
First Submitted That Met QC Criteria
March 29, 2023
First Posted (Actual)
April 3, 2023
Study Record Updates
Last Update Posted (Estimated)
April 26, 2024
Last Update Submitted That Met QC Criteria
April 24, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- NMD670-02-0001
- 2022-002301-24 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Spinal Muscular Atrophy
-
Marco CapogrossoRoche-GenentechRecruitingSpinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type 4United States
-
Institut de Myologie, FranceInstitut RocheCompletedType 2 Spinal Muscular Atrophy | Type 3 Spinal Muscular AtrophyBelgium, France, Germany
-
Marco CapogrossoRoche-GenentechNot yet recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy 4United States
-
Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
-
Hoffmann-La RocheRecruitingSpinal Muscular Atrophy (SMA)Belgium, United States, Croatia, Japan, Netherlands, Spain, Canada, Poland, United Kingdom, Italy, Portugal, Australia
-
Northwell HealthCompletedAdult Spinal Muscular AtrophyUnited States
-
Hugh McMillanFamilies of Spinal Muscular Atrophy; Gwendolyn Strong FoundationTerminatedSpinal Muscular Atrophy (SMA)Canada
-
AveXis, Inc.United BioSource, LLCRecruitingSpinal Muscular Atrophy (SMA)Japan, United States, Korea, Republic of, Israel, Greece, Ireland, Portugal, Russian Federation, Taiwan
-
Hoffmann-La RocheAssociation Française contre les Myopathies (AFM), ParisCompletedSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type III Non AmbulantGermany, Italy, France, Belgium, Poland, Netherlands, United Kingdom
-
Istanbul Medipol University HospitalIstanbul UniversityRecruitingNeuromuscular Diseases | Spinal Muscular Atrophy Type 3Turkey
Clinical Trials on Placebo
-
SamA Pharmaceutical Co., LtdUnknownAcute Bronchitis | Acute Upper Respiratory Tract InfectionKorea, Republic of
-
National Institute on Drug Abuse (NIDA)CompletedCannabis UseUnited States
-
AstraZenecaParexel; Spandauer Damm 130; 14050; Berlin, GermanyCompletedMale Subjects With Type II Diabetes (T2DM)Germany
-
Heptares Therapeutics LimitedCompletedPharmacokinetics | Safety IssuesUnited Kingdom
-
GlaxoSmithKlineCompletedPulmonary Disease, Chronic ObstructiveUnited Kingdom, Netherlands
-
Shijiazhuang Yiling Pharmaceutical Co. LtdXuanwu Hospital, BeijingCompleted
-
GlaxoSmithKlineCompletedInfections, BacterialUnited States
-
ItalfarmacoCompletedBecker Muscular DystrophyNetherlands, Italy
-
West Penn Allegheny Health SystemCompletedAsthma | Allergic RhinitisUnited States