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ClinicalTrials.gov public records Last synced Oct 17, 2026, 8:29 PM EDT

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Showing 1–24 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Office of Rare Diseases (ORD) Clear all
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 17, 2026, 8:29 PM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

NCT00005898
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, fludarabine, methylprednisolone
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
0 Years to 34 Years
Enrollment
30 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 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Completed No phase listed Observational

Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes

NCT00004351
View directory record Official record
Conditions
Williams Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Shprintzen Syndrome, Smith-Magenis Syndrome, DiGeorge Syndrome, Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
0 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia

NCT00005891
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
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 17, 2026, 8:29 PM EDT
Completed Phase 2 Interventional

Phase II Study of High-Dose Cyclophosphamide in Patients With Refractory Pemphigus

NCT00010413
View directory record Official record
Conditions
Pemphigus
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 70 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2008 · Synced Oct 17, 2026, 8:29 PM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Completed No phase listed Observational

CEGIR 7807: Validation of Online Cohort of EGID Patients Enrolled in RDCRN CEGIR Contact Registry

NCT03382678
View directory record Official record
Conditions
Eosinophilic Esophagitis
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
0 Years and older
Enrollment
187 participants
Timeline
2018 – 2020
U.S. locations
2
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

NCT00005892
View directory record Official record
Conditions
Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
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 17, 2026, 8:29 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 17, 2026, 8:29 PM EDT
Withdrawn No phase listed Observational

Examining the Spasmodic Dysphonia Diagnosis and Assessment Procedure (SD-DAP) for Measuring Symptom Change

NCT02061943
View directory record Official record
Conditions
Spasmodic Dysphonia, Laryngeal Dystonia
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Atlanta, Georgia • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 30, 2016 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Phase 2 Interventional Results available

Abatacept for Treating Adults With Giant Cell Arteritis and Takayasu's Arteritis

NCT00556439
View directory record Official record
Conditions
Takayasu's Arteritis, Giant Cell Arteritis
Interventions
Abatacept, Placebo
Drug
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
15 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Baltimore, Maryland • Boston, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2018 · Synced Oct 17, 2026, 8:29 PM EDT
Recruiting No phase listed Observational

One-Time DNA Study for Vasculitis

NCT01241305
View directory record Official record
Conditions
Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss), Giant Cell Arteritis, Granulomatosis With Polyangiitis (Wegener's), Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
7 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 17, 2026, 8:29 PM 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 6, 2012 · Synced Oct 17, 2026, 8:29 PM EDT
Not listed Not applicable Interventional

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

NCT00006056
View directory record Official record
Conditions
Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 55 Years
Enrollment
40 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 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Phase 2 Interventional

Phase II Double-Blind, Placebo-Controlled Study of the Reinforcing Effects of Alprazolam in Patients With Anxiety

NCT00004373
View directory record Official record
Conditions
Anxiety Disorder, Panic Disorder
Interventions
alprazolam
Drug
Lead sponsor
National Institute on Drug Abuse (NIDA)
NIH
Eligibility
18 Years to 50 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Completed No phase listed Observational

Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

NCT00315406
View directory record Official record
Conditions
Polyarteritis Nodosa
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
107 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 17, 2026, 8:29 PM EDT
Not listed Phase 3 Interventional

The Assessment of Prednisone In Remission Trial (TAPIR) - Patient Centric Approach

NCT01933724
View directory record Official record
Conditions
Granulomatosis With Polyangiitis, Wegener Granulomatosis, Vasculitis
Interventions
5 mg prednisone, 0 mg prednisone
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 29, 2024 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Not applicable Interventional

Examining Genetic Differences Among People With 21-Hydroxylase Deficiency

NCT00542841
View directory record Official record
Conditions
21-hydroxylase Deficiency
Interventions
Hydrocortisone withdrawal
Procedure
Lead sponsor
Maria I. New
Other
Eligibility
18 Years to 50 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 14, 2015 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Phase 2 Interventional

Oxandrolone Compared With a Placebo on Growth Rate in Girls With Growth Hormone-Treated Turner's Syndrome

NCT00004275
View directory record Official record
Conditions
Turner's Syndrome
Interventions
growth hormone, oxandrolone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
10 Years to 14 Years · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 10, 2008 · Synced Oct 17, 2026, 8:29 PM EDT
Terminated Phase 1 Interventional

Effect of Potassium and Acetazolamide on People With Andersen-Tawil Syndrome

NCT00839501
View directory record Official record
Conditions
Andersen-Tawil Syndrome
Interventions
Potassium, Acetazolamide
Dietary Supplement · Drug
Lead sponsor
University of Rochester
Other
Eligibility
10 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 20, 2012 · Synced Oct 17, 2026, 8:29 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 17, 2026, 8:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome

NCT02991807
View directory record Official record
Conditions
PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
Interventions
RAD001, Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
5 Years to 45 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 17, 2025 · Synced Oct 17, 2026, 8:29 PM EDT
Completed Not applicable Interventional

Pilot Study of Cyclophosphamide in Patients With Life-Threatening Systemic Lupus Erythematosus or Antiphospholipid Antibody Syndrome

NCT00010400
View directory record Official record
Conditions
Systemic Lupus Erythematosus, Antiphospholipid Antibody Syndrome
Interventions
Cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 70 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 8:29 PM EDT
Suspended Phase 2 Interventional Accepts healthy volunteers

Phase II Study of Stereotypes and Mental Retardation: Neurobiological Basis

NCT00004300
View directory record Official record
Conditions
Stereotyped Behavior, Mental Retardation
Interventions
bromocriptine, sertraline hydrochloride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
U.S. locations
1
States / cities
Morganton, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 6, 2010 · Synced Oct 17, 2026, 8:29 PM EDT