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ClinicalTrials.gov public records Last synced Oct 8, 2026, 2:04 PM EDT

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Showing 25–48 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Office of Rare Diseases (ORD) Clear all
Not listed Phase 1 Interventional

Phase I Study of Immune Ablation and CD34+ Peripheral Blood Stem Cell Support in Patients With Systemic Lupus Erythematosus

NCT00017641
View directory record Official record
Conditions
Systemic Lupus Erythematosus
Interventions
anti-thymocyte globulin, cyclophosphamide, filgrastim, CD34+ Peripheral Blood Stem Cell Reinfusion
Drug · Procedure
Lead sponsor
Northwestern Memorial Hospital
Other
Eligibility
0 Years to 59 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
2
States / cities
Chicago, Illinois • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Genetic Risk Factors for Spina Bifida and Anencephaly

NCT00031122
View directory record Official record
Conditions
Spina Bifida, Anencephaly
Interventions
No Intervention
Other
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
1,100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2012
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 5, 2010 · Synced Oct 8, 2026, 2:04 PM EDT
Completed No phase listed Observational

The Dystonia Coalition Natural History and Biospecimen Repository for Isolated Dystonias

NCT01373424
View directory record Official record
Conditions
Dystonia
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
3,265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2020
U.S. locations
34
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Fountain Valley, California + 26 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2020 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Not applicable Interventional

Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

NCT00010361
View directory record Official record
Conditions
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Interventions
cyclosporine, fludarabine, mycophenolate mofetil
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 9, 2014 · Synced Oct 8, 2026, 2:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Apparent Mineralocorticoid Excess Syndrome

NCT00474942
View directory record Official record
Conditions
Apparent Mineralocorticoid Excess Syndrome
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 11, 2015 · Synced Oct 8, 2026, 2:04 PM EDT
Completed No phase listed Observational

Determining Disease Activity Biomarkers in Individuals With Giant Cell Arteritis

NCT00315497
View directory record Official record
Conditions
Temporal Arteritis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
50 Years and older
Enrollment
426 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
7
States / cities
Boston, Massachusetts • Rochester, Minnesota • Cleveland, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2022 · Synced Oct 8, 2026, 2:04 PM EDT
Not listed No phase listed Observational

Identifying Characteristics of Bone Marrow Failure Syndromes

NCT00315419
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal, Red-Cell Aplasia, Pure, Purpura, Thrombocytopenic, Leukemia, Lymphocytic
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
11 Years and older
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2010 · Synced Oct 8, 2026, 2:04 PM 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 8, 2026, 2:04 PM EDT
Recruiting Phase 2 Interventional

A Randomized Multicenter Study for Isolated Skin Vasculitis

NCT02939573
View directory record Official record
Conditions
Primary Cutaneous Vasculitis, Cutaneous Polyarteritis Nodosa, IgA Vasculitis, Henoch-Schönlein Purpura
Interventions
Colchicine, Dapsone, Azathioprine
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
12
States / cities
Kansas City, Kansas • Boston, Massachusetts • Rochester, Minnesota + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Phase 2 Interventional

Phase II Study of High-Dose Cyclophosphamide in Patients With Severe Autoimmune Hematologic Disease

NCT00010387
View directory record Official record
Conditions
Anemia, Hemolytic, Autoimmune, Felty Syndrome, Purpura, Thrombocytopenic, Autoimmune Diseases
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2008 · Synced Oct 8, 2026, 2:04 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations

NCT02461446
View directory record Official record
Conditions
PTEN, ASD, Autism, Macrocephaly, PTEN Hamartoma Tumor Syndrome
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Months and older
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
5
States / cities
Los Angeles, California • Stanford, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 8, 2026, 2:04 PM EDT
Not listed Phase 1Phase 2 Interventional

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

NCT00319878
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Sirolimus, Cyclosporine
Drug
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
21 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2008 · Synced Oct 8, 2026, 2:04 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome

NCT02461420
View directory record Official record
Conditions
Phelan-McDermid Syndrome, Autism Spectrum Disorder, Intellectual Disability
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Months and older
Enrollment
207 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
5
States / cities
Stanford, California • Chicago, Illinois • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Sirolimus in LAM

NCT00414648
View directory record Official record
Conditions
Lymphangioleiomyomatosis
Interventions
Sirolimus, Placebo
Drug
Lead sponsor
University of Cincinnati
Other
Eligibility
18 Years and older · Female only
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
10
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 2, 2023 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Not applicable Interventional

Randomized Study of Midodrine, an Alpha Adrenergic Agonist, in Patients With Neurally Mediated Syncope

NCT00004479
View directory record Official record
Conditions
Syncope
Interventions
midodrine
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

NCT00694525
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
20 Years to 35 Years · Female only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2009 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Phase 2 Interventional

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
arginine butyrate, epoetin alfa
Drug
Lead sponsor
Boston University
Other
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
6
States / cities
Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

NCT00005893
View directory record Official record
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Phase 1 Interventional

Phase I Pilot Study of Total-Body Irradiation, Anti-Thymocyte Globulin and Cyclophosphamide Followed By Syngeneic or Autologous Peripheral Blood Stem Cell Transplantation in Patients With Multiple Sclerosis

NCT00014755
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
anti-thymocyte globulin, cyclophosphamide, filgrastim, prednisone, peripheral blood stem cell transplantation, irradiation
Drug · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 60 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
6
States / cities
Duarte, California • Denver, Colorado • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Recruiting Phase 4 Interventional

Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis

NCT00004317
View directory record Official record
Conditions
Toxoplasmosis
Interventions
Leucovorin calcium, Pyrimethamine, Spiramycin, Sulfadiazine
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2030
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 14, 2009 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Effect of Estrogen on Mental and Social Functioning in Girls With Turner's Syndrome

NCT00004274
View directory record Official record
Conditions
Turner's Syndrome
Interventions
Estrogen
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 12 Years · Female only
Enrollment
260 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1990
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 2:04 PM EDT
Completed No phase listed Observational

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

NCT00375089
View directory record Official record
Conditions
Prader-Willi Syndrome, Obesity
Interventions
Group 1, Group 2
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 60 Years
Enrollment
392 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
4
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2014 · Synced Oct 8, 2026, 2:04 PM EDT
Completed Phase 1Phase 2 Interventional

Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

NCT00004485
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
cyclosporine, fludarabine, mycophenolate mofetil, Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 16 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
2
States / cities
Oakland, California • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 10, 2008 · Synced Oct 8, 2026, 2:04 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
Conditions
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions
Not listed
Lead sponsor
Andrea Gropman
Other
Eligibility
18 Years to 60 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2015 · Synced Oct 8, 2026, 2:04 PM EDT