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ClinicalTrials.gov public records Last synced Oct 17, 2026, 10:38 PM EDT

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Showing 1129–1152 of 1,710 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 4 Interventional Results available

A Study to Optimize Growth Hormone Dosing in Children With Chronic Kidney Disease by Measuring IGF-1 Levels in Blood

NCT00212758
View directory record Official record
Conditions
Kidney Failure, Chronic, Growth Hormone Deficiency
Interventions
Nutropin AQ
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
8
States / cities
Loma Linda, California • Los Angeles, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced Oct 17, 2026, 10:38 PM EDT
Terminated Phase 3 Interventional

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

NCT00545597
View directory record Official record
Conditions
Adrenomyeloneuropathy, Adrenoleukodystrophy
Interventions
Lorenzo's oil
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 2, 2012 · Synced Oct 17, 2026, 10:38 PM EDT
Terminated Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 17, 2026, 10:38 PM EDT
Recruiting No phase listed Observational

Pulmonary Embolism and Right-to-Left Shunts

NCT07519876
View directory record Official record
Conditions
Pulmonary Embolism (PE), Right-to-Left Shunt, Stroke, Atrial Septal Defects, Patent Foramen Ovale (PFO), Pulmonary Arteriovenous Malformation
Interventions
Not listed
Lead sponsor
Tufts Medical Center
Other
Eligibility
18 Years and older
Enrollment
256 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 17, 2026, 10:38 PM EDT
Terminated No phase listed Observational

Natural History Clinical Study in Adult PKU

NCT04768348
View directory record Official record
Conditions
Phenylketonurias, PKU
Interventions
Not listed
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2023 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

NCT00975689
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl Cysteine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Months and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2013 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Not applicable Interventional

Movement-2-Music: Lakeshore Examination of Activity, Disability, and Exercise Response Study

NCT03797378
View directory record Official record
Conditions
Spinal Cord Injuries, Traumatic Brain Injury, Spina Bifida, Cerebral Palsy, Stroke, Parkinson Disease, Multiple Sclerosis
Interventions
eM2M
Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 70 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 3, 2025 · Synced Oct 17, 2026, 10:38 PM EDT
Recruiting No phase listed Observational

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2033
U.S. locations
12
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 17, 2026, 10:38 PM EDT
Completed No phase listed Observational

Optic Discs in Children With Cerebral Palsy

NCT00769197
View directory record Official record
Conditions
Cerebral Palsy
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
1 Day to 17 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Apr 29, 2009 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 3 Interventional Results available

Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD)

NCT01418352
View directory record Official record
Conditions
Tourette's Disorder
Interventions
Aripiprazole, Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 17 Years
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
13
States / cities
Dothan, Alabama • San Francisco, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2021 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 1 Interventional

Seattle-PAP Bubble Nasal CPAP and Work of Breathing

NCT02210026
View directory record Official record
Conditions
Respiratory Distress Syndrome In Premature Infants, Bronchopulmonary Dysplasia, Newborn Primary Sleep Apnea
Interventions
Seattle-PAP
Device
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
6 Hours to 72 Hours
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 14, 2016 · Synced Oct 17, 2026, 10:38 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 17, 2026, 10:38 PM EDT
Terminated Phase 4 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

NCT01526785
View directory record Official record
Conditions
Pompe Disease
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Year and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
51
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2015 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 3 Interventional

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
14
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2009 · Synced Oct 17, 2026, 10:38 PM EDT
Not listed No phase listed Observational

Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)

NCT06194539
View directory record Official record
Conditions
Fertility Issues
Interventions
Not listed
Lead sponsor
Bar-Chama, Natan, M.D.
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
75 participants
Timeline
2024 – 2026
U.S. locations
1
States / cities
Cresskill, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 8, 2024 · Synced Oct 17, 2026, 10:38 PM EDT
Withdrawn Phase 2 Interventional

Study Evaluating The Safety And Efficacy Of PF-03654746 In Adult Subjects With Tourette's Syndrome

NCT01475383
View directory record Official record
Conditions
Tourette's Syndrome
Interventions
PF-03654746, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 17, 2026, 10:38 PM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 17, 2026, 10:38 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT02935283
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
MRI, Behavioral
Procedure · Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 25, 2024 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Effects of Delayed Cord Clamp and/or Indomethacin on Preterm Infant Brain Injury

NCT02221219
View directory record Official record
Conditions
Intraventricular Hemorrhage, Periventricular Leukomalacia, Brain Injury, Renal Injury
Interventions
Indomethacin, delay in umbilical cord clamp at birth, placebo infusion, immediate cord clamp at birth
Drug · Procedure
Lead sponsor
Hong Huang
Other
Eligibility
24 Weeks to 30 Weeks
Enrollment
256 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Nov 23, 2021 · Synced Oct 17, 2026, 10:38 PM EDT
Approved For Marketing No phase listed Expanded access

Copper Histidinate Treatment for Menkes Disease

NCT04074512
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Not applicable Interventional Results available

The Effect of Video Game Exercise on Dynamic Balance and Gait in Individuals With Huntington's Disease

NCT01735981
View directory record Official record
Conditions
Huntington's Disease
Interventions
Video game exercise using Dance Dance Revolution, hand-held video game
Other
Lead sponsor
Anne Kloos
Other
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 9, 2021 · Synced Oct 17, 2026, 10:38 PM EDT
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2025 · Synced Oct 17, 2026, 10:38 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 17, 2026, 10:38 PM EDT