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ClinicalTrials.gov public records Last synced Oct 18, 2026, 2:01 AM EDT

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Showing 241–264 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Completed Phase 2 Interventional Results available

Surgery and Heated Chemotherapy for Adrenocortical Carcinoma

NCT01833832
View directory record Official record
Conditions
Adrenocortical Carcinoma, Peritoneal Carcinomatosis
Interventions
Cisplatin, Cytoreductive surgery, sodium thiosulfate
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 30, 2019 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting No phase listed Observational

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978
View directory record Official record
Conditions
Duchene Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ITF Therapeutics LLC
Industry
Eligibility
6 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
9
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting No phase listed Observational

Rare Kidney Stone Consortium Patient Registry

NCT00588562
View directory record Official record
Conditions
Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
0 Years to 100 Years
Enrollment
730 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2028
U.S. locations
3
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Phase 2 Interventional

A Study for Patients With Complicated Skin and Skin Structure Infections

NCT00514527
View directory record Official record
Conditions
Staphylococcal Skin Infections, Wounds and Injuries, Abscess, Cellulitis, Streptococcal Infections
Interventions
oritavancin
Drug
Lead sponsor
Targanta Therapeutics Corporation
Industry
Eligibility
18 Years and older
Enrollment
294 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
33
States / cities
Mobile, Alabama • Anaheim, California • Buena Park, California + 29 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2008 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Genomic Uniformed-Screening Against Rare Disease In All Newborns

NCT05990179
View directory record Official record
Conditions
Early Onset Genetic Conditions With Near Complete Penetrance
Interventions
Genome sequencing-based newborn screening
Other
Lead sponsor
Columbia University
Other
Eligibility
1 Day to 1 Month
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT01894165
View directory record Official record
Conditions
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Interventions
ALXN1101, Placebo
Drug
Lead sponsor
Origin Biosciences
Industry
Eligibility
18 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 19, 2019 · Synced Oct 18, 2026, 2:01 AM EDT
Terminated Phase 3 Interventional

Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated Hemolytic Uremic Syndrome

NCT00004465
View directory record Official record
Conditions
Hemolytic Uremic Syndrome
Interventions
SYNSORB Pk, Placebo
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
6 Months to 18 Years
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
26
States / cities
Denver, Colorado • Wilmington, Delaware • Atlanta, Georgia + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2017 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Assessing Pulmonary Changes in HSCT

NCT07823036
View directory record Official record
Conditions
HSCT, Respiratory, Gas Exchange Impairment
Interventions
Hyperpolarized 129 Xenon
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years and older
Enrollment
550 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2029
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Vestibular Dysfunction In Adult Patients With Panic Disorder With or Without Agoraphobia

NCT00004367
View directory record Official record
Conditions
Anxiety Disorder, Panic Disorder
Interventions
Clinical vestibular tests
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years
Enrollment
165 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2000
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Pediatric Vasculitis Initiative

NCT02006134
View directory record Official record
Conditions
Wegeners Granulomatosis (Granulomatosis With Polyangiitis), Microscopic Polyangiitis, Eosinophilic Granulomatosis With Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Primary CNS Vasculitis, Unclassified Vasculitis
Interventions
Not listed
Lead sponsor
University of British Columbia
Other
Eligibility
Up to 20 Years
Enrollment
1,600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2025
U.S. locations
10
States / cities
San Francisco, California • Gainesville, Florida • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2023 · Synced Oct 18, 2026, 2:01 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Hepatic Glucose Production and De Novo Lipogenesis in Patients With Cystic Fibrosis

NCT00014781
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 30 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 12, 2009 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Phase 3 Interventional Results available

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

NCT04966741
View directory record Official record
Conditions
Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 5 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Aurora, Colorado • New York, New York • Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting No phase listed Observational

Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy

NCT07556913
View directory record Official record
Conditions
Pregnancy Related
Interventions
Elafibranor
Drug
Lead sponsor
Ipsen
Industry
Eligibility
Female only
Enrollment
3 participants
Timeline
2026 – 2039
U.S. locations
1
States / cities
Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting No phase listed Observational

Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases (EchoNet-Screening)

NCT05139797
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH screening for cardiac amyloidosis
Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Terminated Phase 3 Interventional Results available

Phase III Randomized Study of Lucinactant in Full Term Newborn Infants With Meconium Aspiration Syndrome

NCT00004500
View directory record Official record
Conditions
Meconium Aspiration
Interventions
Lucinactant, Standard Care
Drug · Other
Lead sponsor
Windtree Therapeutics
Industry
Eligibility
Up to 48 Hours
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
Warrington, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2012 · Synced Oct 18, 2026, 2:01 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Total Energy Expenditure in Infants and Children With Moderate to Severe Cystic Fibrosis

NCT00006273
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 10 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1996
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:01 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Investigating Phenotypic, Epigenetic, and NeuroGenetic Traits in Rare and Ultra-rare Neurodevelopmental Disorders (Project PENGUIN)

NCT07329257
View directory record Official record
Conditions
Baker Gordon Syndrome, Rare Neurodevelopmental Conditions, Rare Neurogenetic Conditions, Syt-1 Disorder, Epilepsy, Seizure, Genetic Mutations, Autism in Children, Developmental Delay (Disorder)
Interventions
No Intervention: Observational Cohort
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
Up to 99 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Not applicable Interventional

Randomized Study of Midodrine, an Alpha Adrenergic Agonist, in Patients With Neurally Mediated Syncope

NCT00004479
View directory record Official record
Conditions
Syncope
Interventions
midodrine
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:01 AM 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 18, 2026, 2:01 AM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Not applicable Interventional

Randomized Study of Recombinant Human Growth Hormone in Patients on Chronic Hemodialysis or Peritoneal Dialysis

NCT00004429
View directory record Official record
Conditions
Kidney Failure, Chronic
Interventions
growth hormone
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2005
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Randomized, Placebo-Controlled Study of Capsaicin for Interstitial Cystitis and Vulvar Vestibulitis

NCT00004316
View directory record Official record
Conditions
Interstitial Cystitis, Vulvar Diseases
Interventions
capsaicin, Placebo
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years and older · Female only
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2007
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 4, 2025 · Synced Oct 18, 2026, 2:01 AM EDT
Completed Phase 2 Interventional Results available

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

NCT02418455
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Day to 5 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Oct 18, 2026, 2:01 AM EDT