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ClinicalTrials.gov public records Last synced Sep 5, 2026, 10:34 AM EDT

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Showing 1–24 of 409 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Rare Diseases Clear all
Not listed No phase listed Observational

Studies in Porphyria I: Characterization of Enzyme Defects

NCT00004331
View directory record Official record
Conditions
Porphyria
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1992
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

NCT01197378
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine Bitartrate Delayed-release Capsules
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced Sep 5, 2026, 10:34 AM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Not applicable Interventional

Effect of Dialysis Dose and Membrane Flux in Maintenance Hemodialysis

NCT00004285
View directory record Official record
Conditions
End Stage Renal Disease
Interventions
Standard dose, low flux hemodialysis, Standard dose, high flux hemodialysis, High dose, low flux hemodialysis, High dose, high flux hemodialysis
Device
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years to 80 Years
Enrollment
1,846 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2001
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 13, 2017 · Synced Sep 5, 2026, 10:34 AM EDT
Completed No phase listed Observational

Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders

NCT03967743
View directory record Official record
Conditions
Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease, Development, Infant, Development, Child
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 4 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Terminated Phase 2 Interventional Results available

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00004418
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
glyceryl trierucate/glyceryl trioleate
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Months to 8 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2014
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Sep 5, 2026, 10:34 AM EDT
Completed No phase listed Observational

COV2Base-A Rare Disease by COVID Study

NCT04564274
View directory record Official record
Conditions
Chronic Health Conditions, Older Age is Associated With Increased Pathogenicity
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Month and older
Enrollment
1,748 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 19, 2023 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Not applicable Interventional

Post-Approval Study of the Guidant Carotid Stent Systems and Embolic Protection Systems: CAPTURE

NCT00180492
View directory record Official record
Conditions
Atherosclerotic Disease, Carotid
Interventions
RX ACCULINK(TM) Carotid Stent System and RX ACCUNET(TM) Embolic Protection System
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Santa Clara, California
Source: ClinicalTrials.gov public record
Updated Oct 28, 2008 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting No phase listed Observational

International Rare Brain Tumor Registry

NCT05697874
View directory record Official record
Conditions
Astroblastoma, BCOR ITD Sarcoma, CNS Sarcoma, Unclassified Tumor, Malignant
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
Up to 45 Years
Enrollment
5,800 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
6
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting Phase 1 Interventional

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355
View directory record Official record
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Not applicable Interventional

Study of Human Botulism Immunoglobulin in Infants With Botulism

NCT00004401
View directory record Official record
Conditions
Infant Botulism, Botulism
Interventions
botulism immune globulin
Drug
Lead sponsor
California Department of Health Services
Other
Eligibility
Up to 1 Year
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998
U.S. locations
1
States / cities
Berkeley, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting No phase listed Observational

Nephrotic Syndrome Study Network

NCT01209000
View directory record Official record
Conditions
Minimal Change Disease (MCD), Membranous Nephropathy, Glomerulosclerosis, Focal Segmental
Interventions
Kidney Biopsy
Procedure
Lead sponsor
University of Michigan
Other
Eligibility
Up to 80 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2030
U.S. locations
39
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 26 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Phase 2 Interventional

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
arginine butyrate, epoetin alfa
Drug
Lead sponsor
Boston University
Other
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
6
States / cities
Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2005 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Human Biospecimen Procurement Protocol: Biorepository to Support Translational Research to Identify Disease Mechanism(s)

NCT02543996
View directory record Official record
Conditions
Undiagnosed Diseases, Cardiovascular Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2050
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Active, not recruiting Phase 2 Interventional

A Study Evaluating the Efficacy and Safety of Biomarker-Driven Therapies in Patients With Persistent or Recurrent Rare Epithelial Ovarian Tumors

NCT04931342
View directory record Official record
Conditions
Ovarian Cancer
Interventions
Ipatasertib, Cobimetinib, Trastuzumab Emtansine, Atezolizumab, Bevacizumab, Paclitaxel, Giredestrant, Abemaciclib, Inavolisib, Palbociclib, Letrozole, Olaparib, Luteinizing Hormone-Releasing Hormone (LHRH) Agonists, Cyclophosphamide
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older · Female only
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
8
States / cities
San Francisco, California • St Louis, Missouri • Charlotte, North Carolina + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

NCT02670694
View directory record Official record
Conditions
Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 18 Years
Enrollment
804 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
9
States / cities
Birmingham, Alabama • Irvine, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2016 · Synced Sep 5, 2026, 10:34 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess Zipalertinib Versus Placebo in Participants With Early Stage NSCLC With Uncommon EGFR Mutations, Following Complete Tumor Resection

NCT07128199
View directory record Official record
Conditions
Lung Cancer, Adjuvant, Post-surgical, EGFR, Exon 20, Early Stage Lung Cancer, Uncommon EGFR Mutations, NSCLC, Early Stage
Interventions
TAS6417, Zipalertinib Matching-placebo
Drug
Lead sponsor
Taiho Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
30
States / cities
Anchorage, Alaska • Duarte, California • Irvine, California + 26 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed No phase listed Observational

Study of Prevalence and Clinical Phenotype in Patients With Glucocorticoid-Remediable Aldosteronism

NCT00004354
View directory record Official record
Conditions
Hyperaldosteronism
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 5, 2026, 10:34 AM EDT
Completed No phase listed Observational

Next Generation to Identify Genetic Causes of Disease in Patients Participating in NICHD Clinical Protocols

NCT01375543
View directory record Official record
Conditions
Genetic Predisposition
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 5, 2020 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting No phase listed Observational

Monogenic Kidney Stone - Genetic Testing

NCT03305835
View directory record Official record
Conditions
Rare Kidney Stone Diseases
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Not applicable Interventional

Study of Recombinant Human Insulin-Like Growth Factor I in Patients With Severe Insulin Resistance

NCT00004419
View directory record Official record
Conditions
Insulin Resistance, Hyperglycemia
Interventions
insulin-like growth factor I
Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
14 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 5, 2026, 10:34 AM EDT
Recruiting Phase 2 Interventional

Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors

NCT01356290
View directory record Official record
Conditions
Medulloblastoma Recurrent, Ependymoma Recurrent, ATRT Recurrent, Rare CNS Tumor Recurrent
Interventions
Bevacizumab, Thalidomide, Celecoxib, Fenofibric acid, Etoposide, Cyclophosphamide, Etoposide phosphate, Cytarabine, Temozolomide (TMZ), Irinotecan
Drug
Lead sponsor
Medical University of Vienna
Other
Eligibility
Up to 19 Years
Enrollment
232 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • Boston, Massachusetts • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2026 · Synced Sep 5, 2026, 10:34 AM EDT
Completed Phase 3 Interventional

Phase III Randomized Study of Oral Acyclovir in Infants With Herpes Simplex Virus Infection Limited to Skin, Eyes, and Mouth

NCT00006135
View directory record Official record
Conditions
Herpes Simplex
Interventions
acyclovir
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 28 Days
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
25
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 5, 2026, 10:34 AM EDT