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ClinicalTrials.gov public records Last synced Oct 17, 2026, 12:26 AM EDT

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Showing 1–24 of 164 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Muscular Atrophy, Progressive Clear all
Completed No phase listed Observational

Measuring Levels of SMN in Blood Samples of SMA Patients

NCT00061607
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 6, 2017 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting No phase listed Observational

Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

NCT04174157
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Prospective observational registry, Zolgensma
Other · Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2038
U.S. locations
50
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 45 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2025 · Synced Oct 17, 2026, 12:26 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 9, 2025 · Synced Oct 17, 2026, 12:26 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer

NCT03437941
View directory record Official record
Conditions
Metastatic Castration-Resistant Prostate Cancer
Interventions
Exicorilant, Enzalutamide, Placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Scottsdale, Arizona • Detroit, Michigan • Basking Ridge, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting Phase 3 Interventional

A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)

NCT07723248
View directory record Official record
Conditions
Metastatic Castration Resistant Prostate Cancer
Interventions
Rinzimetostat, Darolutamide, Enzalutamide, Docetaxel
Drug
Lead sponsor
ORIC Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
11
States / cities
Coral Springs, Florida • Jeffersonville, Indiana • Holland, Michigan + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Active, not recruiting Not applicable Interventional

COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

NCT05035823
View directory record Official record
Conditions
Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injuries, Stroke, Lacunar, Stroke Brainstem, Cervical Spinal Cord Injury, Spinal Muscular Atrophy, Tetraplegic; Paralysis, Quadriplegia/Tetraplegia
Interventions
Motor Neuroprosthesis (MNP)
Device
Lead sponsor
Synchron Medical, Inc.
Industry
Eligibility
21 Years to 75 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
3
States / cities
Buffalo, New York • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs

NCT07336446
View directory record Official record
Conditions
Prostate Cancer
Interventions
AZD9750, AZD5305
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
2
States / cities
Duarte, California • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting No phase listed Observational

Clinical Procedures to Support Research in ALS

NCT03489278
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2034
U.S. locations
11
States / cities
Irvine, California • Palo Alto, California • San Francisco, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting No phase listed Observational

A Registry of Cases of Spinal Muscular Atrophy

NCT07805278
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
30 participants
Timeline
2026 – 2033
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Pilot Study of Biomarkers for Spinal Muscular Atrophy

NCT00756821
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
2 Years to 12 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
16
States / cities
Birmingham, Alabama • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2012 · Synced Oct 17, 2026, 12:26 AM EDT
Completed Phase 1 Interventional

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

NCT01780246
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 17, 2026, 12:26 AM EDT
Terminated Phase 2 Interventional

A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.

NCT03196375
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
FLX-787-ODT (orally disintegrating tablet), Placebo ODT
Drug
Lead sponsor
Flex Pharma, Inc.
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
35
States / cities
Scottsdale, Arizona • Sacramento, California • San Francisco, California + 29 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2018 · Synced Oct 17, 2026, 12:26 AM EDT
Completed Phase 2 Interventional Results available

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

NCT03032172
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
6 Months to 60 Years
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
4
States / cities
Palo Alto, California • Orlando, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 17, 2026, 12:26 AM EDT
Completed No phase listed Observational

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

NCT02124057
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2022 · Synced Oct 17, 2026, 12:26 AM EDT
Completed No phase listed Observational

Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy

NCT05518773
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 3, 2025 · Synced Oct 17, 2026, 12:26 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Mechanisms of Cell Death in Spinal Muscular Atrophy

NCT01754441
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2020
U.S. locations
2
States / cities
Wilmington, Delaware • Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 21, 2020 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting Phase 2 Interventional

A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

NCT07287982
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
16
States / cities
Little Rock, Arkansas • San Diego, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Completed Phase 3 Interventional Results available

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277
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 26, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

NCT02292537
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 12 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
11
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 17, 2026, 12:26 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
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 13, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Terminated Phase 1 Interventional Results available

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT03381729
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
6 Months to 60 Months
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Stanford, California • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 17, 2026, 12:26 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 17, 2026, 12:26 AM EDT