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ClinicalTrials.gov public records Last synced Jun 26, 2026, 5:17 AM EDT

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Showing 1–24 of 1,939 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 3 Interventional

Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome

NCT02179151
View directory record Official record
Conditions
Prader-Willi Syndrome, Obesity
Interventions
ZGN-440 for Injectable Suspension, ZGN-440 Placebo for Injectable Suspension
Drug
Lead sponsor
Zafgen, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
15
States / cities
Sacramento, California • San Diego, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2017 · Synced Jun 26, 2026, 5:17 AM EDT
Enrolling by invitation Phase 2 Interventional

Sirolimus for Leigh Syndrome

NCT06843811
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Jun 26, 2026, 5:17 AM EDT
Recruiting No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 1 Interventional

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

NCT00739505
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
2
States / cities
St Louis, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 28, 2019 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 5, 2017 · Synced Jun 26, 2026, 5:17 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jun 26, 2026, 5:17 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Mutations Associated With Parkinson s Disease

NCT01547832
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Jun 26, 2026, 5:17 AM EDT
Terminated Not applicable Interventional Results available

Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer

NCT00726830
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 23, 2020 · Synced Jun 26, 2026, 5:17 AM EDT
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Not applicable Interventional Results available

Effect of Chronic Incretin-based Therapy in Cystic Fibrosis

NCT01879228
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Insufficiency
Interventions
Sitagliptin, Placebo
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2022 · Synced Jun 26, 2026, 5:17 AM EDT
Recruiting No phase listed Observational

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

NCT05588167
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Jun 26, 2026, 5:17 AM EDT
Recruiting No phase listed Observational

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

NCT04883905
View directory record Official record
Conditions
Acute Hepatic Porphyria
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
13
States / cities
Birmingham, Alabama • Gilbert, Arizona • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jun 26, 2026, 5:17 AM EDT
Completed No phase listed Observational

Natural History Studies of Mucopolysaccharidosis III

NCT02037880
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA, Mucopolysaccharidosis Type IIIB
Interventions
Lumbar puncture, Magnetic Resonance Imaging (MRI) of the brain
Procedure · Device
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
2 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 15, 2015 · Synced Jun 26, 2026, 5:17 AM EDT
Terminated Phase 1 Interventional

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

NCT04110496
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 3 Interventional

A Randomized, Controlled Study of Intravenous Ganciclovir Therapy for Peripheral Cytomegalovirus Retinitis in Patients With AIDS

NCT00000688
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
22
States / cities
San Diego, California • San Francisco, California • San Mateo, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2011 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
8 Months and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
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 Jun 26, 2026, 5:17 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

NCT01459302
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease, Lou Gehrigs Disease, Familial Disease, Amyotrophic Lateral Sclerosis, Sporadic, Muscular Dystrophy, Miyoshi Myopathy, Amyotrophic Lateral Sclerosis With Dementia
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2024
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jun 26, 2026, 5:17 AM EDT
Terminated Phase 2 Interventional Results available

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
7 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2016 · Synced Jun 26, 2026, 5:17 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 6, 2024 · Synced Jun 26, 2026, 5:17 AM EDT
Completed No phase listed Observational

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324
View directory record Official record
Conditions
Fabry Disease
Interventions
Interview
Other
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Jun 26, 2026, 5:17 AM EDT
Completed Phase 1 Interventional

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00048620
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Nov 2, 2006 · Synced Jun 26, 2026, 5:17 AM EDT