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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
54
UCLA David Geffen School Of Medicine - Neurology
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Distance walked (meters)
Time frame: Baseline to day 21
Change from baseline in muscle strength versus placebo
Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)
Time frame: Baseline to day 21
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
percentage change in distance walked in 6th minute compared to 1st minute
Time frame: 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
Time frame: Baseline to day 21
Change from baseline in jitter versus placebo
Jitter (micro seconds) assessed with single fiber EMG
Time frame: Baseline to day 21
Change from baseline in blocking versus placebo
Blocking (%) assessed with single fiber EMG
Time frame: Baseline to day 21
Incidence of treatment emergent adverse events
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of serious treatment emergent adverse events
Summarised per treatment
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Los Angeles, California, United States
Stanford University Medical Center
Palo Alto, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
The Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore, Maryland, United States
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Rare Disease Research - Raleigh-Durham
Hillsborough, North Carolina, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
Neurology Rare Disease Center
Denton, Texas, United States
UZ Leuven - Neurochirurgie Campus Gasthuisberg
Leuven, Belgium
...and 15 more locations
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of clinically significant ECG abnormalities
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior
Summarised per treatment
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations
Summarised per treatment
Time frame: Over 21 days of dosing