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ClinicalTrials.gov public records Last synced May 21, 2026, 7:44 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 148 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Spinal Muscular Atrophy (SMA) Clear all
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
SMA - Spinal Muscular Atrophy, Gene Therapy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 180 Days
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
16
States / cities
Los Angeles, California • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1986 – 2018
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 6, 2018 · Synced May 21, 2026, 7:44 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
Up to 6 Months
Enrollment
53 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
15
States / cities
Davis, California • Los Angeles, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated May 3, 2018 · Synced May 21, 2026, 7:44 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2021 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced May 21, 2026, 7:44 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
Interventions
Apalutamide, Cetrelimab
Drug · Biological
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 14, 2025 · Synced May 21, 2026, 7:44 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
610 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2026
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 19, 2025 · Synced May 21, 2026, 7:44 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 120 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2022 · Synced May 21, 2026, 7:44 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Breast Cancer
Interventions
suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
Drug
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
8
States / cities
San Francisco, California • Boynton Beach, Florida • Orlando, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2014 · Synced May 21, 2026, 7:44 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Valproic Acid (VPA), Placebo
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 60 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 5, 2016 · Synced May 21, 2026, 7:44 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Sacramento, California • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
25 participants
Timeline
2018 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 19, 2023 · Synced May 21, 2026, 7:44 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced May 21, 2026, 7:44 PM EDT
Recruiting Phase 2 Interventional
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
ARGX-119 IV, Placebo IV
Biological · Other
Lead sponsor
argenx
Industry
Eligibility
5 Years to 17 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
17
States / cities
Little Rock, Arkansas • San Diego, California • Stanford, California + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced May 21, 2026, 7:44 PM EDT
Suspended Phase 1 Interventional
View directory record Official record
Conditions
Prostatic Neoplasm
Interventions
EZN-4176
Drug
Lead sponsor
Enzon Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 17, 2012 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced May 21, 2026, 7:44 PM EDT
Recruiting Phase 4 Interventional
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
10
States / cities
Little Rock, Arkansas • Madera, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced May 21, 2026, 7:44 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 21, 2026, 7:44 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year to 6 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced May 21, 2026, 7:44 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease, PMA - Progressive Muscular Atrophy, PBP - Progressive Bulbar Palsy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
14
States / cities
Loma Linda, California • Los Angeles, California • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 21, 2026, 7:44 PM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
4-aminopyridine, Placebo
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 50 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 2, 2024 · Synced May 21, 2026, 7:44 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
4,805 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced May 21, 2026, 7:44 PM EDT