Clinical Trials Sponsored by Office of Rare Diseases Research (ORDR)

Total 362 results

Office of Rare Diseases (ORD)

Unknown

Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers

Adrenal Hyperplasia, Congenital

United States, Brazil, France
Office of Rare Diseases (ORD)

Unknown

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

Adrenal Hyperplasia, Congenital

United States
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network

Unknown

Identifying Characteristics of Bone Marrow Failure Syndromes

Myelodysplastic Syndromes | Purpura, Thrombocytopenic | Anemia, Aplastic | Red-Cell Aplasia, Pure | Bone Marrow Failure Syndromes | Hemoglobinuria, Paroxysmal | Leukemia, Lymphocytic

United States
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network

Unknown

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

Anemia, Aplastic

United States
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network

Unknown

Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
Boston University

Completed

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

Beta-Thalassemia

United States, United Kingdom
Duke University

Completed

Study of Axial Mobility Exercises in Parkinson Disease

Parkinson Disease
University of Minnesota

Unknown

Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

Fanconi's Anemia
University of Utah

Completed

Phase II Study of Growth Hormone in Children With Cystic Fibrosis

Cystic Fibrosis

United States
University of Utah

Completed

Study of Hepatic Glucose Production and De Novo Lipogenesis in Patients With Cystic Fibrosis

Cystic Fibrosis

United States
University of Minnesota

Completed

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

Fanconi's Anemia

United States
Johns Hopkins University

Completed

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

Pemphigus

United States
Northwestern Memorial Hospital

Completed

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

Multiple Sclerosis

United States
House Ear Institute

Unknown

NF2 Natural History Consortium

Meningioma | Neurofibromatosis 2 | Schwannoma, Vestibular

United States
Johns Hopkins University

Completed

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

Systemic Lupus Erythematosus | Antiphospholipid Antibody Syndrome

United States
Northwestern Memorial Hospital

Unknown

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

Systemic Lupus Erythematosus

United States
Northwestern Memorial Hospital

Unknown

Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia

Aplastic Anemia | Graft Versus Host Disease

United States
Johns Hopkins University

Completed

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

Autoimmune Diseases | Purpura, Thrombocytopenic | Anemia, Hemolytic, Autoimmune | Felty Syndrome

United States
University of California, Los Angeles
Rare Diseases Clinical Research Network; Office of Rare Diseases (ORD)

Completed

Characteristics of Episodic Ataxia Syndrome

Cerebellar Diseases | Episodic Ataxia Syndrome

United States, Canada, United Kingdom
Fairview University Medical Center

Terminated

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Immunologic Deficiency Syndromes | Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Graft Versus Host Disease | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | Virus-Associated... and other conditions

United States
Fairview University Medical Center

Unknown

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

Mucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell Disease

United States
Fred Hutchinson Cancer Center

Completed

Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

Sickle Cell Anemia

United States
Fred Hutchinson Cancer Center

Completed

Phase II Pilot Study of Cyclophosphamide and Rabbit Anti-Thymocyte Globulin as Salvage Therapy in Patients With Diffuse Systemic Sclerosis

Systemic Sclerosis

United States
Fairview University Medical Center

Completed

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

Fanconi's Anemia

United States
Dana-Farber Cancer Institute

Unknown

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

Fanconi's Anemia

United States
Fairview University Medical Center

Completed

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

Myelodysplastic Syndromes | Leukemia, Lymphocytic, Acute | Fanconi's Anemia | Leukemia, Nonlymphocytic, Acute

United States
Roswell Park Cancer Institute

Completed

Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia

Aplastic Anemia

United States
Fred Hutchinson Cancer Center

Completed

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

Metabolism, Inborn Errors | Granulomatous Disease, Chronic

United States
Fairview University Medical Center

Completed

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

Neutropenia | Sickle Cell Anemia | Red-Cell Aplasia, Pure | Thalassemia Major

United States
Fairview University Medical Center

Unknown

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

Rheumatoid Arthritis | Systemic Lupus Erythematosus | Giant Cell Arteritis | Wegener's Granulomatosis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Purpura, Schoenlein-Henoch | Juvenile Rheumatoid Arthritis | Takayasu Arteritis | Pure Red Cell Aplasia | Graft Versus Host Disease | Anemia, Hemolytic, Autoimmune and other conditions

United States
Fairview University Medical Center

Unknown

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

Hemophagocytic Lymphohistiocytosis | Graft Versus Host Disease | Chediak-Higashi Syndrome | Virus-Associated Hemophagocytic Syndrome | X-Linked Lymphoproliferative Syndrome | Familial Erythrophagocytic Lymphohistiocytosis

United States
Fred Hutchinson Cancer Center

Completed

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

Multiple Sclerosis

United States
University of Alabama at Birmingham

Completed

Phase II Study of Rituximab in Patients With Immune Thrombocytopenic Purpura

Purpura, Thrombocytopenic, Idiopathic

United States
University of Rochester
National Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaborators

Completed

Characteristics of Andersen-Tawil Syndrome

Andersen-Tawil Syndrome | Andersen Syndrome

United States, Canada, Italy, United Kingdom
Richard Barohn, MD
Rare Diseases Clinical Research Network; Office of Rare Diseases (ORD)

Completed

Characteristics of Nondystrophic Myotonias

Myotonic Disorders | Myotonia Congenita | Nondystrophic Myotonias

Canada, United States, United Kingdom
Herbert Irving Comprehensive Cancer Center

Completed

Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis

Amyloidosis

United States
National Center for Research Resources (NCRR)
Mayo Clinic

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of 3,4-Diaminopyridine for Demyelinating Polyneuropathy

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita

Epidermolysis Bullosa Acquisita
National Center for Research Resources (NCRR)
Yale University

Completed

Phase III Randomized, Placebo-Controlled, Crossover Study of Soy Protein Isolate for Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia
National Center for Research Resources (NCRR)
Northwestern University

Completed

Randomized Placebo-Controlled Study of Aerobic Exercise and Resistance Training Plus Megestrol Acetate for HIV-Wasting

HIV Infections | Acquired Immunodeficiency Syndrome | HIV Wasting Syndrome
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Study of Lactulose and Circadian Rhythms in Patients With Cirrhosis

Hepatic Encephalopathy | Cirrhosis | Portal Hypertension
National Center for Research Resources (NCRR)
Emory University

Completed

Phase II Study of Pallidotomy for Parkinson Disease

Parkinson Disease
National Center for Research Resources (NCRR)
Yale University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Guanfacine for Tourette Syndrome and Attention Deficit Hyperactivity Disorder

Tourette Syndrome
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Study of Long-Term Dehydroepiandrosterone for Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
National Center for Research Resources (NCRR)
Stanford University

Completed

A Maintenance Extension of Phase I Pilot Study of Chimeric Anti-CD4 Antibody M-T412 in Patients With Multiple Sclerosis

Multiple Sclerosis
National Center for Research Resources (NCRR)
Yale University

Completed

Phase II Pilot Study of Octreotide, a Somatostatin Octapeptide Analog, for Gastrointestinal Hemorrhage in Hormone-Refractory Hereditary Hemorrhagic Telangiectasia and Senile Ectasia

Hereditary Hemorrhagic Telangiectasia | Ectasia
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II/III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone in Women With Mild to Moderate Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
National Center for Research Resources (NCRR)
Northwestern University

Completed

Study of Genotype and Phenotype Expression in Congenital Nephrogenic Diabetes Insipidus

Diabetes Insipidus, Nephrogenic
National Center for Research Resources (NCRR)
Northwestern University

Completed

Study of Pituitary Size and Function in Familial Dwarfism of Sindh

Growth Hormone Deficiency
National Center for Research Resources (NCRR)
Scripps Clinic

Completed

Phase II Pilot Study of Cladribine (2-Chlorodeoxyadenosine; 2-CdA) for Early Stage Primary Sclerosing Cholangitis

Cholangitis, Sclerosing

Clinical Trials Sponsored by Office of Rare Diseases Research (ORDR)

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