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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Rare Diseases Clear all
Completed No phase listed Observational

Rare CNS Tumors Outcomes &Risk

NCT03251989
View directory record Official record
Conditions
High Grade Meningioma, Ependymoma, Medulloblastoma, PNET, Primary CNS Sarcoma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
326 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Completed Phase 2 Interventional Results available

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

NCT00303329
View directory record Official record
Conditions
Anemia, Hemosiderosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
5
States / cities
Oakland, California • Stanford, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 8, 2011 · Synced Jul 22, 2026, 2:09 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 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Completed Not applicable Interventional

Rare Group Problem Management Plus

NCT06548022
View directory record Official record
Conditions
Anxiety, Depressive Symptoms, Post-traumatic Stress Disorder, Problems Psychosocial
Interventions
Rare Group Problem Management Plus
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
18 Years to 99 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 29, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Jul 22, 2026, 2:09 AM EDT
Not listed No phase listed Observational

International Registry for Severe Chronic Neutropenia

NCT00004342
View directory record Official record
Conditions
Neutropenia
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
3 Months and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1994
U.S. locations
5
States / cities
Boston, Massachusetts • Worcester, Massachusetts • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2020 · Synced Jul 22, 2026, 2:09 AM EDT
Terminated No phase listed Observational

Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency

NCT00005098
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
160 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 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Completed Phase 2 Interventional

Phase II Study of Rituximab in Patients With Immune Thrombocytopenic Purpura

NCT00005652
View directory record Official record
Conditions
Purpura, Thrombocytopenic, Idiopathic
Interventions
rituximab
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Sep 8, 2008 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting No phase listed Observational

Rare and Atypical Diabetes Network

NCT05544266
View directory record Official record
Conditions
Diabetes Mellitus, Diabetes Mellitus Progression, Glucose Intolerance, Glucose Metabolism Disorders, Metabolic Disease, Endocrine; Complications, Endocrine System Diseases
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
13
States / cities
Aurora, Colorado • Chicago, Illinois • Indianapolis, Indiana + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2025 · Synced Jul 22, 2026, 2:09 AM EDT
Withdrawn No phase listed Observational

Finding Genes for Rare Diseases

NCT02724995
View directory record Official record
Conditions
Rare Diseases
Interventions
Not listed
Lead sponsor
University of Kentucky
Other
Eligibility
1 Minute to 101 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Oct 25, 2017 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting No phase listed Observational

OCT in Rare Chorioretinal Diseases

NCT02141308
View directory record Official record
Conditions
Retinal Artery Occlusions, Polypoidal Choroidal Vasculopathy, Retinal Arterial Macroaneurysm, Juxtafoveal Telangiecasia, Central Serous Chorioretinopathy
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
7 Years to 100 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 11, 2024 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting No phase listed Observational

Rare Glycogen Storage Diseases Natural History Study

NCT06795152
View directory record Official record
Conditions
Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
Interventions
No intervention
Other
Lead sponsor
Duke University
Other
Eligibility
0 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting No phase listed Observational

CCHS Secure Health-hub Advancing Research Efforts (CCHS SHARE)

NCT06554275
View directory record Official record
Conditions
Congenital Central Hypoventilation Syndrome
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
Not listed
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 15, 2025 · Synced Jul 22, 2026, 2:09 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 Jul 22, 2026, 2:09 AM EDT
Active, not recruiting Phase 2 Interventional

Talimogene Laherparepvec and Nivolumab in Treating Patients With Refractory Lymphomas or Advanced or Refractory Non-melanoma Skin Cancers

NCT02978625
View directory record Official record
Conditions
Anaplastic Large Cell Lymphoma, ALK-Negative, Anaplastic Large Cell Lymphoma, ALK-Positive, Apocrine Carcinoma, Carcinoma Arising From Cylindroma, Carcinoma Arising From Spiradenoma, Digital Papillary Adenocarcinoma, Endocrine Mucin-Producing Sweat Gland Carcinoma, Extramammary Paget Disease, Extraocular Sebaceous Carcinoma, Hidradenocarcinoma, Malignant Sweat Gland Neoplasm, Merkel Cell Carcinoma, Microcystic Adnexal Carcinoma, NK-Cell Lymphoma, Unclassifiable, Papillary Adenocarcinoma, Porocarcinoma, Primary Cutaneous Mucinous Carcinoma, Recurrent Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Recurrent T-Cell Non-Hodgkin Lymphoma, Refractory Anaplastic Large Cell Lymphoma, Refractory Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Refractory Merkel Cell Carcinoma, Refractory Mycosis Fungoides, Refractory Primary Cutaneous T-Cell Non-Hodgkin Lymphoma, Refractory Skin Squamous Cell Carcinoma, Refractory T-Cell Non-Hodgkin Lymphoma, Sezary Syndrome, Signet Ring Cell Adenocarcinoma, Skin Adenoid Cystic Carcinoma, Skin Adnexal Carcinoma, Skin Basal Cell Carcinoma, Skin Basosquamous Cell Carcinoma, Skin Keratoacanthoma, Skin Squamous Cell Carcinoma, Squamoid Eccrine Ductal Carcinoma, Squamous Cell Carcinoma of Unknown Primary, Sweat Gland Carcinoma, Trichilemmal Carcinoma, Vulvar Squamous Cell Carcinoma
Interventions
Biopsy Procedure, Biospecimen Collection, Computed Tomography, Laboratory Biomarker Analysis, Nivolumab, Positron Emission Mammography, Talimogene Laherparepvec
Procedure · Other · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
36
States / cities
Los Angeles, California • Orange, California • Pasadena, California + 28 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 5, 2012 · Synced Jul 22, 2026, 2:09 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 25, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Suspended Phase 2 Interventional

A Phase II Clinical Trial to Evaluate Safety and Efficacy of XmAb20717 in Advanced Rare Cancers

NCT05337735
View directory record Official record
Conditions
Microsatellite High Cancers, Peritoneal Mesothelioma, Extrapulmonary High Grade, Neuroendocrine Carcinomas, Cervical Carcinoma, Hodgkin's Lymphoma, Pleural Mesothelioma, Small Cell Lung Cancer
Interventions
XmAb20717
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Jul 22, 2026, 2:09 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Energy Requirements in Critically Ill Newborns

NCT00006274
View directory record Official record
Conditions
Respiratory Distress Syndrome
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
24 Hours to 120 Hours
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1997
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Completed Phase 2 Interventional

Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

NCT00004323
View directory record Official record
Conditions
Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, mesna, methotrexate, methylprednisolone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Not listed Not applicable Interventional

Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases

NCT00006055
View directory record Official record
Conditions
Purpura, Schoenlein-Henoch, Graft Versus Host Disease, Anemia, Hemolytic, Autoimmune, Rheumatoid Arthritis, Churg-Strauss Syndrome, Hypersensitivity Vasculitis, Wegener's Granulomatosis, Systemic Lupus Erythematosus, Giant Cell Arteritis, Pure Red Cell Aplasia, Juvenile Rheumatoid Arthritis, Polyarteritis Nodosa, Autoimmune Thrombocytopenic Purpura, Takayasu Arteritis
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, methylprednisolone, prednisone, Autologous Peripheral Blood Stem Cell Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
1 Year to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Study of Metabolic Effects of Pregnancy in Women With Cystic Fibrosis

NCT00014768
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
glucose, insulin, leucine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older · Female only
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 2:09 AM EDT
Completed Phase 2 Interventional Results available

A Study Osimertinib in Patients With Stage 4 Non-small Cell Lung Cancer With Uncommon EGFR Mutations

NCT03434418
View directory record Official record
Conditions
Non Small Cell Lung Cancer
Interventions
osimertinib
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Durham, North Carolina • Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 16, 2023 · Synced Jul 22, 2026, 2:09 AM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 22, 2026, 2:09 AM EDT