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ClinicalTrials.gov public records Last synced Oct 17, 2026, 10:20 PM EDT

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Showing 193–216 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Active, not recruiting Phase 2 Interventional

Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors

NCT02574728
View directory record Official record
Conditions
Cancer
Interventions
Sirolimus, Celecoxib, Etoposide, Cyclophosphamide
Drug
Lead sponsor
Emory University
Other
Eligibility
12 Months to 30 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
6
States / cities
Phoenix, Arizona • Wilmington, Delaware • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Completed No phase listed Observational

Pilot Study of Familial Nonsyndromal Mondini Dysplasia

NCT00004336
View directory record Official record
Conditions
Mondini Dysplasia
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 10:20 PM EDT
Recruiting Not applicable Interventional

Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors

NCT07851636
View directory record Official record
Conditions
Rare CNS Tumor, FOXR2-activated, BCOR ITD Sarcoma, CNS Sarcoma, PLAG-altered Tumors, Mesenchymal Tumor
Interventions
Personalized Treatment Recommendation
Other
Lead sponsor
Adriana Fonseca
Other
Eligibility
Up to 45 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Recruiting Phase 1 Interventional

Effects of Midodrine and Droxidopa on Splanchnic Capacitance in Autonomic Failure

NCT02897063
View directory record Official record
Conditions
Autonomic Failure, Pure Autonomic Failure, Multiple System Atrophy, Parkinson Disease, Orthostatic Hypotension
Interventions
Droxidopa, Midodrine, Placebo
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
40 Years to 80 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Not applicable Interventional

Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks

NCT00004330
View directory record Official record
Conditions
Porphyria
Interventions
luteinizing hormone-releasing factor
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1987
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 10:20 PM 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 25, 2015 · Synced Oct 17, 2026, 10:20 PM EDT
Completed No phase listed Observational

Rare Genetic Disorders of the Breathing Airways

NCT00323167
View directory record Official record
Conditions
Kartagener Syndrome, Cystic Fibrosis, Pseudohypoaldosteronism, Primary Ciliary Dyskinesia
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
7
States / cities
Palo Alto, California • Denver, Colorado • Bethesda, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2022 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia

NCT00004492
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
clotrimazole, hydroxyurea
Drug
Lead sponsor
University of North Carolina
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 17, 2026, 10:20 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Alpha-2 Adrenergic Receptor Dysfunction in Regional Lipoatrophy

NCT00004329
View directory record Official record
Conditions
Lipodystrophy
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years · Female only
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 1 Interventional

Seneca Valley Virus-001 and Cyclophosphamide in Treating Young Patients With Relapsed or Refractory Neuroblastoma, Rhabdomyosarcoma, or Rare Tumors With Neuroendocrine Features

NCT01048892
View directory record Official record
Conditions
Adrenocortical Carcinoma, Gastrointestinal Carcinoid Tumor, Kidney Cancer, Neuroblastoma, Retinoblastoma, Sarcoma
Interventions
Seneca Valley virus-001, cyclophosphamide, laboratory biomarker analysis, pharmacological study
Biological · Drug · Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
3 Years to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
18
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2014 · Synced Oct 17, 2026, 10:20 PM EDT
Active, not recruiting Phase 2 Interventional

A Phase 2 Study of T-DXd in Patients With Selected HER2 Expressing Tumors

NCT04482309
View directory record Official record
Conditions
Part 1: Bladder, Biliary Tract, Cervical, Endometrial, Ovarian, Pancreatic Cancer, Rare Tumors, Any Tumor Type Excluding Breast, Gastric, Colorectal Cancer, Part 2: HER2 Expressing/Amplified Solid Tumors Excluding Breast, Gastric, Colorectal Cancer
Interventions
Trastuzumab deruxtecan
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 120 Years
Enrollment
477 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
12
States / cities
Duarte, California • Santa Rosa, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Completed No phase listed Observational

Study of the Pathogenesis and Pathophysiology of Familial Neurohypophyseal Diabetes Insipidus

NCT00004363
View directory record Official record
Conditions
Diabetes Insipidus, Diabetes Insipidus, Neurohypophyseal
Interventions
chlorpropamide, desmopressin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
6 Months to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 2Phase 3 Interventional

Deep Brain Stimulation in Treating Patients With Dystonia

NCT00004421
View directory record Official record
Conditions
Dystonia
Interventions
implanted pulse generator
Device
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years to 75 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 3 Interventional Results available

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

NCT01733316
View directory record Official record
Conditions
Cystinosis
Interventions
RP103, Cystagon®
Drug
Lead sponsor
Amgen
Industry
Eligibility
12 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
5
States / cities
San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 2 Interventional Results available

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

NCT01573754
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Hydroxychloroquine, Phlebotomy
Drug · Procedure
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 17, 2023 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 3 Interventional

Study of Tetrathiomolybdate in Patients With Wilson Disease

NCT00004339
View directory record Official record
Conditions
Wilson Disease
Interventions
tetrathiomolybdate, trientine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
90 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 May 9, 2006 · Synced Oct 17, 2026, 10:20 PM EDT
Completed No phase listed Observational

Humanistic Burden of (FSGS) Focal Segmental Glomerulosclerosis and IgAN (Immunoglobulin A Nephropathy)

NCT05200871
View directory record Official record
Conditions
Focal Segmental Glomerulosclerosis, Immunoglobulin A Nephropathy
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
487 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
4
States / cities
San Francisco, California • Belmar, New Jersey • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2024 · Synced Oct 17, 2026, 10:20 PM 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 Sep 29, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 1Phase 2 Interventional Results available

JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease

NCT05998395
View directory record Official record
Conditions
Kohlmeier Degos Disease With Neurologic Involvement, Kohlmeier-Degos Disease
Interventions
Ruxolitinib
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Not listed Phase 2 Interventional

Phase II Randomized Study of Intravenous Versus Oral Clomipramine in Patients With Obsessive Compulsive Disorder

NCT00004310
View directory record Official record
Conditions
Obsessive-Compulsive Disorder
Interventions
Clomipramine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
2
States / cities
Stanford, California • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 10:20 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 1 Interventional

Phase I Study of Alpha-Melanocyte Stimulating Hormone in Patients With Acute Renal Failure

NCT00004496
View directory record Official record
Conditions
Acute Renal Failure
Interventions
alpha-melanocyte stimulating hormone
Drug
Lead sponsor
University of Texas
Other
Eligibility
18 Years to 80 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2003
U.S. locations
2
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 17, 2026, 10:20 PM 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 24, 2005 · Synced Oct 17, 2026, 10:20 PM EDT
Completed Phase 2 Interventional

Alternate Day Prednisone or Daily Fish Oil Supplements in Patients With Immunoglobulin A Nephropathy

NCT00004448
View directory record Official record
Conditions
IgA Glomerulonephritis
Interventions
omega-3 fatty acids, prednisone, Placebo
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Up to 40 Years
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2007
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 4, 2025 · Synced Oct 17, 2026, 10:20 PM EDT