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ClinicalTrials.gov public records Last synced Oct 18, 2026, 7:35 AM EDT

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Showing 313–336 of 411 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rare Disorder Clear all
Terminated Phase 3 Interventional Accepts healthy volunteers Results available

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Enrolling by invitation No phase listed Observational

The Mayo Clinic Rare and Undiagnosed Disease Hackathon

NCT07714161
View directory record Official record
Conditions
Undiagnosed Diseases, Genetic Diseases, Rare Diseases
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 1 Interventional

Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

NCT00004842
View directory record Official record
Conditions
Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
Interventions
budesonide
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years to 70 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 1999
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 31, 2017 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

NCT01000961
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon® (Cysteamine Bitartrate), Cysteamine Bitartrate Delayed-release Capsules (RP103)
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
3
States / cities
Stanford, California • Atlanta, Georgia • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 18, 2026, 7:35 AM EDT
Recruiting No phase listed Observational

International Rare Histiocytic Disorders Registry (IRHDR)

NCT02285582
View directory record Official record
Conditions
Rare Histiocytic Disorders (RHDs), Erdheim-Chester Disease (ECD), Rosai-Dorfman Disease (RDD), Xanthogranuloma Family (XG), Indeterminate Dendritic Cell Histiocytosis, Malignant Histiocytic Neoplasm (MHN), ALK-positive Histiocytosis, Mixed Histiocytosis (MXH), Multicentric Reticulohistiocytoma (MRH), Necrobiotic Xanthogranuloma (NX)
Interventions
Registry study
Other
Lead sponsor
The Hospital for Sick Children
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
6
States / cities
Birmingham, Alabama • Los Angeles, California • Madera, California + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2025 · Synced Oct 18, 2026, 7:35 AM EDT
Temporarily Not Available No phase listed Expanded access

RAPA-501 Therapy of ALS Expanded Access Protocol

NCT06169176
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
RAPA-501 Autologous T stem cells
Other
Lead sponsor
Rapa Therapeutics LLC
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
10
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Irvine, California + 7 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 1Phase 2 Interventional

Result of tDCS in ASD Children With Comorbidities Like PANDAS, Rare Genetic Diseases or Autoimmune Disorders

NCT06368726
View directory record Official record
Conditions
Attention, Visual Perceptual Weakness, Social Behavior, Fluency Disorder, EEG With Periodic Abnormalities
Interventions
tDCS or Transcranial Direct Current Stimulation, Risperidone, Sham tDCS
Device · Drug · Other
Lead sponsor
Spanish Foundation for Neurometrics Development
Other
Eligibility
6 Years to 11 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational

Evaluation and Treatment of Patients With Retinal Disease

NCT00005784
View directory record Official record
Conditions
Retinal Disease
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
10 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational

Study of the Neurobiology of Tourette Syndrome and Related Disorders

NCT00004325
View directory record Official record
Conditions
Tourette Syndrome, Obsessive Compulsive Disorder
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
12 Years to 45 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1988
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

NCT02116764
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years and older
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Recruiting Not applicable Interventional

TCF-001 TRACK (Target Rare Cancer Knowledge) Study

NCT04504604
View directory record Official record
Conditions
Rare Cancers, Cholangiocarcinoma, Cancer of Unknown Primary Site
Interventions
FoundationOne CDx and FoundationOne Liquid CDx
Diagnostic Test
Lead sponsor
TargetCancer Foundation
Other
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Not applicable Interventional

Randomized Study of Decreased Hyperinsulinemia on the Ovulatory Response to Clomiphene Citrate in Women With Polycystic Ovary Syndrome

NCT00005104
View directory record Official record
Conditions
Polycystic Ovary Syndrome, Hyperinsulinism
Interventions
clomiphene citrate, metformin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 40 Years · Female only
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
5
States / cities
Birmingham, Alabama • Shreveport, Louisiana • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational

T-Cell Project: Prospective Collection of Data in Patients With Peripheral T-Cell Lymphoma

NCT01142674
View directory record Official record
Conditions
Lymphoma, T-Cell, Peripheral
Interventions
Not listed
Lead sponsor
Associazione Angela Serra per la ricerca sul cancro
Other
Eligibility
18 Years and older
Enrollment
1,650 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2018
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2019 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 2 Interventional

Phase II Study of Calcitonin for Tumoral Calcinosis

NCT00004358
View directory record Official record
Conditions
Calcinosis
Interventions
calcitonin
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1992
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 2 Interventional Results available

Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

NCT03013543
View directory record Official record
Conditions
Genetic Obesity, Obesity, Obesity Due to Melanocortin 4 Receptor Deficiency
Interventions
Setmelanotide
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
43
States / cities
Birmingham, Alabama • Chandler, Arizona • Mesa, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2023 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Not applicable Interventional

Randomized Study of Oral Contraceptives or Hormone Replacement Therapy in Women With Systemic Lupus Erythematosus

NCT00006133
View directory record Official record
Conditions
Systemic Lupus Erythematosus
Interventions
estradiol, ethinyl estradiol, medroxyprogesterone, norethindrone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older · Female only
Enrollment
970 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
17
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX

NCT03366337
View directory record Official record
Conditions
IgA Nephropathy, CKD Associated With Type 1 Diabetes, Focal Segmental Glomerulosclerosis, Autosomal Dominant Polycystic Kidney
Interventions
Bardoxolone methyl capsules
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 65 Years
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
30
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2025 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

NCT01880983
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP)
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
7 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2020
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 18, 2026, 7:35 AM EDT
Not listed Phase 2 Interventional

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 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, 7:35 AM EDT
Withdrawn Phase 2 Interventional

Radioactive Drug (177Lu-DOTATATE) for the Treatment of Locally Advanced, Metastatic, or Unresectable Rare Endocrine Cancers

NCT04106843
View directory record Official record
Conditions
Locally Advanced Adrenal Gland Pheochromocytoma, Locally Advanced Paraganglioma, Metastatic Adrenal Gland Pheochromocytoma, Metastatic Paraganglioma, Metastatic Parathyroid Gland Carcinoma, Pituitary Gland Carcinoma, Somatostatin Receptor Positive, Stage III Thyroid Gland Medullary Carcinoma AJCC v8, Stage IV Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVA Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVB Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVC Thyroid Gland Medullary Carcinoma AJCC v8, Unresectable Adrenal Gland Pheochromocytoma, Unresectable Paraganglioma
Interventions
Lutetium Lu 177 Dotatate
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 7, 2020 · Synced Oct 18, 2026, 7:35 AM EDT
Not yet recruiting Not applicable Interventional Accepts healthy volunteers

Evaluation of Impact of AI Assistance on Workload Associated w Preparation of Rare Tumor Case Repts

NCT06026098
View directory record Official record
Conditions
Cognitive Burden, Cognitive Symptom
Interventions
Artificial Intelligence
Behavioral
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 18, 2026, 7:35 AM EDT
Completed Phase 2 Interventional

Phase II Study of Midodrine for Neurogenic Orthostatic Hypotension

NCT00004268
View directory record Official record
Conditions
Orthostatic Hypotension
Interventions
midodrine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT
Recruiting Phase 2 Interventional

The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

NCT04372615
View directory record Official record
Conditions
Autoimmune Encephalitis, Encephalitis
Interventions
Inebilizumab, Placebo
Drug
Lead sponsor
University of Utah
Other
Eligibility
12 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
35
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 28 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2025 · Synced Oct 18, 2026, 7:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Gonadotropin-Releasing Hormone Pulse Frequency in Sexual Maturation and in the Menstrual Cycle

NCT00004335
View directory record Official record
Conditions
Hypogonadism, Precocious Puberty
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
7 Years to 35 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1993
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 7:35 AM EDT