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ClinicalTrials.gov public records Last synced Oct 18, 2026, 2:43 AM EDT

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Showing 265–288 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Rare and Undiagnosed Disease Research Biorepository

NCT04703179
View directory record Official record
Conditions
Undiagnosed Disease, Rare Diseases
Interventions
Genetic test evaluation
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting No phase listed Observational

Stakeholders of Rare Diseases Informing Values In Neuroethics

NCT07314736
View directory record Official record
Conditions
Rare Disorder, Disorder, Neurologic
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
385 participants
Timeline
2026 – 2031
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting No phase listed Observational

Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation

NCT06258902
View directory record Official record
Conditions
Pregnancy Related
Interventions
Not listed
Lead sponsor
Ipsen
Industry
Eligibility
Female only
Enrollment
20 participants
Timeline
2023 – 2032
U.S. locations
1
States / cities
Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Not applicable Interventional Results available

Study of an Implantable Functional Neuromuscular Stimulation System for Patients With Spinal Cord Injuries

NCT00004445
View directory record Official record
Conditions
Spinal Cord Injury
Interventions
Surgery, IRS-8
Procedure · Device
Lead sponsor
Case Western Reserve University
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2016
U.S. locations
2
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 20, 2025 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Not applicable Interventional

Randomized Study of L-Baclofen in Patients With Refractory Trigeminal Neuralgia

NCT00004431
View directory record Official record
Conditions
Trigeminal Neuralgia
Interventions
L-baclofen
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 18, 2026, 2:43 AM EDT
Not listed Phase 1 Interventional

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

NCT00006127
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

The Effects of a Nitrate Supplementation on Erectile Function

NCT06213077
View directory record Official record
Conditions
Erectile Dysfunction
Interventions
Nitric Oxide, Tadalafil, Placebo
Dietary Supplement · Drug · Other
Lead sponsor
RHA Investments Ltd
Industry
Eligibility
40 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 19, 2024 · Synced Oct 18, 2026, 2:43 AM EDT
Completed No phase listed Observational

Natural History of the Progression of X-Linked Retinitis Pigmentosa

NCT04926129
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
Other: Assessments
Other
Lead sponsor
NightstaRx Ltd, a Biogen Company
Other
Eligibility
7 Years and older
Enrollment
201 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
10
States / cities
Phoenix, Arizona • Los Angeles, California • San Francisco, California + 7 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Completed No phase listed Observational

Hippocampal Complex Volume and Memory Dysfunction in Cushing's Syndrome

NCT00004326
View directory record Official record
Conditions
Cushing's Syndrome
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
15 Years to 80 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1994
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Completed No phase listed Observational

Study of Genetic Anomalies of Complement Related Proteins in Patients With IgA Glomerulonephritis

NCT00004305
View directory record Official record
Conditions
IGA Glomerulonephritis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Not applicable Interventional Results available

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

NCT01802567
View directory record Official record
Conditions
Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
Interventions
Guided Therapy- Pediatric Gene Analysis Platform
Device
Lead sponsor
Giselle Sholler
Other
Eligibility
13 Months and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2024
U.S. locations
13
States / cities
Phoenix, Arizona • San Diego, California • Hartford, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting No phase listed Observational

The KidneyCARE (Community Access to Research Equity) Study

NCT05497518
View directory record Official record
Conditions
Chronic Kidney Disease, Dialysis, Kidney Transplant
Interventions
Observational study - registry
Other
Lead sponsor
Tufts Medical Center
Other
Eligibility
18 Years to 120 Years
Enrollment
50,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2070
U.S. locations
1
States / cities
Danville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 13, 2025 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Not applicable Interventional

Study of the Pharmacokinetics of Mycophenolate Mofetil in Patients Who Have Undergone Orthotopic Liver Transplantation

NCT00007059
View directory record Official record
Conditions
Graft Versus Host Disease
Interventions
mycophenolate mofetil
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
19 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting Phase 1 Interventional

Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms

NCT04541082
View directory record Official record
Conditions
Central Nervous System Neoplasms, Glioblastoma, Gliosarcoma, Adult, Anaplastic Oligodendroglioma, Anaplastic Astrocytoma, Pilocytic Astrocytoma, Oligodendroglioma, Gliomatosis Cerebri, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Diffuse Midline Glioma, H3 K27M-Mutant, Ependymoma, Ependymoma, Anaplastic, Medulloblastoma, Teratoid Rhabdoid Tumor, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Anaplastic Meningioma, Atypical Meningioma, Choroid Plexus Neoplasms, Pineal Tumor, Diffuse Astrocytoma, Glial Tumor
Interventions
ONC206
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 18, 2026, 2:43 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 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Completed No phase listed Observational

Cross-sectional Characterization of Idiopathic Bronchiectasis

NCT01264055
View directory record Official record
Conditions
Infection, Bronchial Diseases, Respiratory Tract Diseases
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
5
States / cities
Stanford, California • Denver, Colorado • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting No phase listed Observational

Study to Investigate Genetic Causes of Severe Early Childhood Onset Obesity.

NCT01998750
View directory record Official record
Conditions
Obesity
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Up to 80 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 18, 2026, 2:43 AM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

NCT00004307
View directory record Official record
Conditions
Amino Acid Metabolism, Inborn Errors
Interventions
Protein and calorie controlled diet, Ornithine transcarbamylase vector
Behavioral · Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
6 Months to 64 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Phase 2 Interventional

Phase II Randomized Study of Collagenase in Patients With Residual Type Dupuytren's Disease

NCT00004409
View directory record Official record
Conditions
Dupuytren's Contracture
Interventions
collagenase
Drug
Lead sponsor
State University of New York
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2004
U.S. locations
2
States / cities
Palo Alto, California • Stony Brook, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Phase 1 Interventional

Phase I Study of Vasoactive Intestinal Peptide in Patients With Acute Respiratory Distress Syndrome and Sepsis

NCT00004494
View directory record Official record
Conditions
Sepsis, Respiratory Distress Syndrome, Respiratory Distress Syndrome, Adult
Interventions
vasoactive intestinal peptide
Drug
Lead sponsor
Stony Brook University
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
2
States / cities
Northport, New York • Stony Brook, New York
Source: ClinicalTrials.gov public record
Updated May 4, 2022 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Evaluation of Vestibular Stimulation to Help Occasional Sleeplessness

NCT00594022
View directory record Official record
Conditions
Insomnia, Sleeplessness, Transient Insomnia
Interventions
Electric stimulation of the Vestibular Nerve - "VirtuSom", Electric stim of the Vestibular Nerve - "VirtuSom" - SHAM
Device
Lead sponsor
Philips Respironics
Industry
Eligibility
21 Years to 50 Years
Enrollment
349 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2008
U.S. locations
6
States / cities
Atlanta, Georgia • New York, New York • Rochester, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2019 · Synced Oct 18, 2026, 2:43 AM EDT
Recruiting No phase listed Observational

Epilepsy Learning Healthcare System (ELHS)

NCT06265103
View directory record Official record
Conditions
Epilepsy, Seizure Disorder, Neurologic Disorder, Rare Diseases
Interventions
Clinical care and quality improvement
Other
Lead sponsor
Epilepsy Foundation of America
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2099
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2024 · Synced Oct 18, 2026, 2:43 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT00872729
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon®, RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 18, 2026, 2:43 AM EDT