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
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.
UCLA David Geffen School Of Medicine - Neurology
Los Angeles, California, United States
RECRUITINGStanford University Medical Center
Palo Alto, California, United States
RECRUITINGUniversity of Colorado Neurology Clinic
Aurora, Colorado, United States
WITHDRAWNUF Fixel Institute for Neurological Diseases
Gainesville, Florida, United States
WITHDRAWNRare Disease Center
Atlanta, Georgia, United States
WITHDRAWNUniversity of Kansas Medical Center
Kansas City, Kansas, United States
RECRUITINGThe Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore, Maryland, United States
RECRUITINGRoy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States
RECRUITINGWashington University School of Medicine
St Louis, Missouri, United States
RECRUITINGRare Disease Research - Raleigh-Durham
Hillsborough, North Carolina, United States
RECRUITING...and 21 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