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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-002301-24 | EudraCT Number |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Experimental drug followed by placebo |
|
| Cohort 2 | Experimental | Placebo followed by experimental drug |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NMD670 | Drug | Tablets |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in 6 minute walk test (6MWT) total distance versus placebo | Distance walked (meters) | Baseline to day 21 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in muscle strength versus placebo | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA David Geffen School Of Medicine - Neurology | Los Angeles | California | 90095 | United States | ||
| Stanford University Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41224659 | Derived | Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025. |
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2-way crossover
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| Drug |
Tablets |
|
percentage change in distance walked in 6th minute compared to 1st minute
| Baseline to day 21 |
| Change from baseline in Revised Hammersmith Scale (RHS) versus placebo | Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms | Baseline to day 21 |
| Change from baseline in jitter versus placebo | Jitter (micro seconds) assessed with single fiber EMG | Baseline to day 21 |
| Change from baseline in blocking versus placebo | Blocking (%) assessed with single fiber EMG | Baseline to day 21 |
| Incidence of treatment emergent adverse events | Summarised per treatment | Over 21 days of dosing |
| Incidence of serious treatment emergent adverse events | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on physical examinations | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on safety laboratory parameters | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant vital signs abnormalities | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant ECG abnormalities | Summarised per treatment | Over 21 days of dosing |
| Incidence of Suicidal Ideation or Suicidal Behavior | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on opthalmological examinations | Summarised per treatment | Over 21 days of dosing |
| Palo Alto |
| California |
| 94304 |
| United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| The Johns Hopkins Medicine, Spinal Muscular Atrophy Center | Baltimore | Maryland | 21287 | United States |
| Roy Blunt NextGen Precision Health Institute | Columbia | Missouri | 65212 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Rare Disease Research - Raleigh-Durham | Hillsborough | North Carolina | 27278 | United States |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | United States |
| Neurology Rare Disease Center | Denton | Texas | 76208 | United States |
| UZ Leuven - Neurochirurgie Campus Gasthuisberg | Leuven | Belgium |
| CHR de la Citadelle - Neurologie | Liège | Belgium |
| Heritage Medical Research Clinic | Calgary | Canada |
| Genge Partners Inc. | Montreal | Canada |
| Aarhus Universitetshospital, Neurologisk Afdeling | Aarhus | Denmark |
| Rigshospitalet - Neurologisk Afdeling | Copenhagen | Denmark |
| Charite - Campus Virchow-Klinikum (CVK) | Berlin | Germany |
| Universitätsklinikum Essen - Klinik Für Neurologie | Essen | Germany |
| Istituto Giannina Gaslini, IRCCS | Genova | Italy |
| Istituto Neurologico C. Besta, Fondazione IRCCS | Milan | Italy |
| Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda | Milan | Italy |
| AOU Città della Salute e della Scienza di Torino | Torino | Italy |
| Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology | Utrecht | Netherlands |
| Hospital Universitari Vall D Hebron | Barcelona | Spain |
| Hospital Materno Infantil La Paz | Madrid | Spain |
| Hospital Universitario y Politécnico La Fe | Valencia | Spain |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
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