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ClinicalTrials.gov public records Last synced Oct 17, 2026, 4:53 PM EDT

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Showing 97–120 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

A Pilot Study of Rapid Haplotyping Procedure for Personalized Dosing of Dichloroacetate (DCA) in Healthy Volunteers

NCT02690285
View directory record Official record
Conditions
Healthy
Interventions
Dichloroacetate (DCA), GSTZ1 haplotyping
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 70 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 31, 2018 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Not applicable Interventional

Randomized Study of Alendronate in Adult Patients With Cystic Fibrosis Related Osteoporosis

NCT00004489
View directory record Official record
Conditions
Osteoporosis, Cystic Fibrosis
Interventions
alendronate sodium, calcium carbonate, cholecalciferol
Drug
Lead sponsor
University of North Carolina
Other
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 17, 2026, 4:53 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Rare Diseases Clinical Research Network: Neurophysiological Correlates

NCT03077308
View directory record Official record
Conditions
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
Interventions
Auditory and Visual Event-related Potentials and EEG
Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 65 Years
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
5
States / cities
Denver, Colorado • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 17, 2026, 4:53 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

The Longitudinal CONQUER Study of Rare Neuroimmunologic Disorders

NCT01623076
View directory record Official record
Conditions
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Transverse Myelitis, Optic Neuritis
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
6 Years and older
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2030
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Not listed No phase listed Observational

Cure Cystinosis International Registry

NCT01327807
View directory record Official record
Conditions
Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
Interventions
Not listed
Lead sponsor
Cystinosis Research Foundation
Other
Eligibility
Not listed
Enrollment
750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2022
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2014 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Phase 3 Interventional Results available

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

NCT03936777
View directory record Official record
Conditions
Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
Interventions
ZX008 (Fenfluramine Hydrochloride)
Drug
Lead sponsor
Zogenix, Inc.
Industry
Eligibility
2 Years and older
Enrollment
412 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
26
States / cities
Tucson, Arizona • Los Angeles, California • San Diego, California + 23 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Phase 2 Interventional Results available

Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers

NCT00141765
View directory record Official record
Conditions
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent
Interventions
Myeloablative Chemotherapy, Stem Cell Rescue
Procedure
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2010
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 20, 2014 · Synced Oct 17, 2026, 4:53 PM EDT
Terminated Phase 2 Interventional Results available

Immune Checkpoint Inhibitor Nivolumab in People With Recurrent Select Rare CNS Cancers

NCT03173950
View directory record Official record
Conditions
Medulloblastoma, Ependymoma, Pineal Region Tumors, Choroid Plexus Tumors, Atypical/Malignant Meningioma
Interventions
Nivolumab, EKG, MRI Brain and/or spine, Body CT Scan, MDASI-BT, MDASI-SP
Drug · Diagnostic Test · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Chicago, Illinois • Bethesda, Maryland • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Terminated Not applicable Interventional

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

NCT00006054
View directory record Official record
Conditions
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
Up to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 15, 2009 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Phase 1 Interventional

Phase I Study of High-Dose Cyclophosphamide and Total Body Irradiation With T Lymphocyte-Depleted Autologous Peripheral Blood Stem Cell or Bone Marrow Rescue in Patients With Multiple Sclerosis

NCT00017628
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
cyclophosphamide, filgrastim, methylprednisolone, Autologous Stem Cell Transplantation
Drug · Procedure
Lead sponsor
Northwestern Memorial Hospital
Other
Eligibility
0 Years to 59 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
2
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Active, not recruiting Phase 1 Interventional

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

NCT03478462
View directory record Official record
Conditions
Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG, Neuroblastoma, Ewing Sarcoma, Rhabdomyosarcoma, Osteosarcoma
Interventions
CLR 131
Drug
Lead sponsor
Cellectar Biosciences, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
6
States / cities
Palo Alto, California • New York, New York • Chapel Hill, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 17, 2026, 4:53 PM 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 Sep 15, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Enrolling by invitation No phase listed Observational

International Registry of Congenital Portosystemic Shunt (IRCPSS)

NCT06041906
View directory record Official record
Conditions
Congenital Portosystemic Shunt, CPSS (Congenital Portosystemic Shunt)
Interventions
Shunt Closure
Procedure
Lead sponsor
Prof. Valérie Mc Lin
Other
Eligibility
1 Day and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
2
States / cities
New Haven, Connecticut • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 18, 2025 · Synced Oct 17, 2026, 4:53 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Pembrolizumab in Treating Patients With Rare Tumors That Cannot Be Removed by Surgery or Are Metastatic

NCT02721732
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Carcinoma of Unknown Primary, Metastatic Adrenal Gland Pheochromocytoma, Metastatic Kidney Medullary Carcinoma, Metastatic Malignant Germ Cell Tumor, Metastatic Malignant Solid Neoplasm, Metastatic Paraganglioma, Metastatic Penile Carcinoma, Metastatic Skin Squamous Cell Carcinoma, Small Cell Carcinoma, Stage III Adrenal Cortex Carcinoma AJCC v7, Stage IV Adrenal Cortex Carcinoma AJCC v7, Stage IV Penile Cancer AJCC v7, Stage IV Renal Cell Cancer AJCC v7, Unresectable Adrenal Gland Pheochromocytoma, Unresectable Paraganglioma, Unresectable Skin Squamous Cell Carcinoma, Unresectable Solid Neoplasm, Vascular Neoplasm
Interventions
Laboratory Biomarker Analysis, Pembrolizumab, Questionnaire Administration
Other · Biological
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Recruiting Phase 2Phase 3 Interventional

Placebo-Controlled Trial of IFx-Hu2.0 Followed By Pembrolizumab In Checkpoint Inhibitor Naïve Participants With Advanced Or Metastatic Merkel Cell Carcinoma

NCT06947928
View directory record Official record
Conditions
Advanced Or Metastatic Merkel Cell Carcinoma
Interventions
IFx-Hu2.0, Placebo, Pembrolizumab
Drug
Lead sponsor
TuHURA Biosciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
18
States / cities
Los Angeles, California • San Francisco, California • Stanford, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Recruiting No phase listed Observational

Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome

NCT05528744
View directory record Official record
Conditions
Genetic Disease, Chopra-Amiel-Gordon Syndrome, CAGS, ANKRD17
Interventions
Observational Study, Sample collection only
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Not applicable Interventional

Study of Fluoxetine in Patients With Depersonalization Disorder

NCT00004446
View directory record Official record
Conditions
Depersonalization Disorder
Interventions
fluoxetine
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years to 65 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 4:53 PM EDT
Recruiting No phase listed Observational

Natural History of Uncommon Dyslipidemias, Rare Lipid Disorders and Unusual Atherosclerotic Conditions

NCT06676046
View directory record Official record
Conditions
Dyslipidemia, Atherosclerosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
10 Years to 90 Years
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
Completed Not applicable Interventional

Study of the Relationship Between Feeding and Late Onset Sepsis and/or Necrotizing Enterocolitis in Low Birth Weight Infants

NCT00005888
View directory record Official record
Conditions
Necrotizing Enterocolitis, Sepsis
Interventions
Fortified Pasteurized Donor Human Milk supplement, Preterm Formula supplement
Behavioral
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 96 Hours
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 4:53 PM EDT
Not listed No phase listed Observational

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

NCT04080050
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
Drug
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
4
States / cities
Columbus, Ohio • Portland, Oregon • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced Oct 17, 2026, 4:53 PM EDT
Terminated No phase listed Observational

An Open Label Navigational Investigation of Molecular Profile-Related Evidence Determining Individualized Cancer Therapy for Patients With Incurable Hematologic Malignancies (I-PREDICT Heme)

NCT03955276
View directory record Official record
Conditions
Hematologic Cancer
Interventions
Molecularly targeted treatment matched to genomic/immunophenotypic tumor profile (chosen by treating physician)
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Feb 15, 2022 · Synced Oct 17, 2026, 4:53 PM EDT
Active, not recruiting Phase 2 Interventional

Nivolumab and Ipilimumab in Treating Patients With Rare Tumors

NCT02834013
View directory record Official record
Conditions
Acinar Cell Carcinoma, Adenoid Cystic Carcinoma, Adrenal Cortical Carcinoma, Adrenal Gland Pheochromocytoma, Anal Canal Neuroendocrine Carcinoma, Anal Canal Undifferentiated Carcinoma, Angiosarcoma, Apocrine Neoplasm, Appendix Mucinous Adenocarcinoma, Bartholin Gland Transitional Cell Carcinoma, Basal Cell Carcinoma, Bladder Adenocarcinoma, Breast Metaplastic Carcinoma, Cervical Adenocarcinoma, Cholangiocarcinoma, Chordoma, Colorectal Squamous Cell Carcinoma, Desmoid Fibromatosis, Endometrial Transitional Cell Carcinoma, Endometrioid Adenocarcinoma, Esophageal Neuroendocrine Carcinoma, Esophageal Undifferentiated Carcinoma, Extrahepatic Bile Duct Carcinoma, Extramammary Paget Disease, Fallopian Tube Adenocarcinoma, Fallopian Tube Transitional Cell Carcinoma, Fibromyxoid Tumor, Gallbladder Carcinoma, Gastric Neuroendocrine Carcinoma, Gastric Squamous Cell Carcinoma, Gastric Undifferentiated Carcinoma, Gastrointestinal Stromal Tumor, Gestational Trophoblastic Tumor, Giant Cell Carcinoma, Human Papillomavirus-Independent Cervical Adenocarcinoma, Clear Cell-Type, Intestinal Neuroendocrine Carcinoma, Intrahepatic Cholangiocarcinoma, Lung Neuroendocrine Tumor, Lung Sarcomatoid Carcinoma, Major Salivary Gland Carcinoma, Malignant Neoplasm of Unknown Primary, Malignant Odontogenic Neoplasm, Malignant Peripheral Nerve Sheath Tumor, Malignant Solid Neoplasm, Malignant Testicular Sex Cord-Stromal Tumor, Metastatic Pituitary Neuroendocrine Tumor, Minimally Invasive Lung Adenocarcinoma, Mixed Mesodermal (Mullerian) Tumor, Mucinous Adenocarcinoma, Mucinous Cystadenocarcinoma, Nasal Cavity Adenocarcinoma, Nasal Cavity Carcinoma, Nasopharyngeal Carcinoma, Nasopharyngeal Low Grade Papillary Adenocarcinoma, Nasopharyngeal Undifferentiated Carcinoma, Oral Cavity Carcinoma, Oropharyngeal Undifferentiated Carcinoma, Ovarian Adenocarcinoma, Ovarian Germ Cell Tumor, Ovarian Mucinous Adenocarcinoma, Ovarian Squamous Cell Carcinoma, Ovarian Transitional Cell Carcinoma, Pancreatic Acinar Cell Carcinoma, Pancreatic Neuroendocrine Carcinoma, Paraganglioma, Paranasal Sinus Adenocarcinoma, Paranasal Sinus Carcinoma, Parathyroid Gland Carcinoma, PEComa, Penile Squamous Cell Carcinoma, Peritoneal Mesothelial Neoplasm, Placental Choriocarcinoma, Primary Peritoneal High Grade Serous Adenocarcinoma, Pseudomyxoma Peritonei, Rare Disorder, Scrotal Squamous Cell Carcinoma, Seminal Vesicle Adenocarcinoma, Seminoma, Serous Cystadenocarcinoma, Small Intestinal Adenocarcinoma, Small Intestinal Squamous Cell Carcinoma, Spindle Cell Neoplasm, Teratoma With Somatic-Type Malignancy, Testicular Non-Seminomatous Germ Cell Tumor, Thyroid Gland Carcinoma, Tracheal Carcinoma, Transitional Cell Carcinoma, Ureter Adenocarcinoma, Ureter Squamous Cell Carcinoma, Urethral Adenocarcinoma, Urethral Squamous Cell Carcinoma, Vaginal Adenocarcinoma, Vaginal Squamous Cell Carcinoma, Not Otherwise Specified, Vulvar Carcinoma
Interventions
Biospecimen Collection, Computed Tomography, Echocardiography Test, Ipilimumab, Magnetic Resonance Imaging, Nivolumab
Procedure · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
798 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1006
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 620 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 17, 2026, 4:53 PM EDT
No Longer Available No phase listed Expanded access

Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access

NCT01797276
View directory record Official record
Conditions
Mitochondrial Enzyme Deficiencies
Interventions
Dichloroacetate
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 8, 2017 · Synced Oct 17, 2026, 4:53 PM EDT