Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Jun 26, 2026, 3:32 AM EDT

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Showing 1–24 of 1,800 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Completed Phase 2Phase 3 Interventional Results available

HEALEY ALS Platform Trial - Regimen D Pridopidine

NCT04615923
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Pridopidine, Matching Placebo
Drug
Lead sponsor
Merit E. Cudkowicz, MD
Other
Eligibility
18 Years and older
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Enrolling by invitation Phase 2 Interventional

Sirolimus for Leigh Syndrome

NCT06843811
Conditions
Leigh Syndrome
Interventions
Sirolimus
Drug
Lead sponsor
Matthew Demczko
Other
Eligibility
6 Months to 55 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
U.S. locations
20
States / cities
Los Angeles, California • Oakland, California • Fort Lauderdale, Florida + 14 more
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Conditions
Amyotrophic Lateral Sclerosis
Interventions
MyoRegulator®
Device
Lead sponsor
PathMaker Neurosystems Inc.
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Charlestown, Massachusetts
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Conditions
Hepatic Encephalopathy
Interventions
lactulose
Drug
Lead sponsor
Hunter Holmes Mcguire Veteran Affairs Medical Center
Federal
Eligibility
18 Years to 75 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Richmond, Virginia
Approved For Marketing No phase listed Expanded access

Copper Histidinate Treatment for Menkes Disease

NCT04074512
Conditions
Menkes Disease
Interventions
copper histidinate
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
0 Years to 6 Years
U.S. locations
20
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 17 more
Terminated No phase listed Observational Accepts healthy volunteers

Mutations Associated With Parkinson s Disease

NCT01547832
Conditions
Parkinson's Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Spartanburg, South Carolina • Houston, Texas
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase, Diet Only
Drug · Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
12 Years to 17 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Amyotrophic Lateral Sclerosis
Interventions
BIIB078, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
12
States / cities
La Jolla, California • Los Angeles, California • Palo Alto, California + 9 more
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Months and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
19 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Charlotte, North Carolina
Completed Phase 1Phase 2 Interventional Results available

Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

NCT02039401
Conditions
Amyotrophic Lateral Sclerosis
Interventions
VM202
Biological
Lead sponsor
Helixmith Co., Ltd.
Industry
Eligibility
21 Years to 75 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Stroke, Hyperglycemia
Interventions
IV glucose insulin and potassium, GIK, standard care
Drug · Other
Lead sponsor
University of Virginia
Other
Eligibility
18 Years and older
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
2
States / cities
Augusta, Georgia • Charlottesville, Virginia
Conditions
Niemann-Pick Disease, Type C
Interventions
arimoclomol, Placebo
Drug
Lead sponsor
ZevraDenmark
Industry
Eligibility
2 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
NeuRx® Diaphragm Pacing System™ (DPS)
Device
Lead sponsor
Barrow Neurological Institute
Other
Eligibility
21 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 21 more
Conditions
Phenylketonurias
Interventions
RTX-134
Drug
Lead sponsor
Rubius Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Aurora, Colorado
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Conditions
Gestational Diabetes, Type 2 Diabetes Mellitus
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
New Haven, Connecticut
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