Clinical Trials Sponsored by Office of Rare Diseases (ORD)

Total 457 results

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)
Northwestern University

Unknown

Study of Novel Types of Familial Diabetes Insipidus

Diabetes Insipidus
National Center for Research Resources (NCRR)
Stanford University

Completed

Pilot Study of Enalapril and Renal Function in Patients With IgA Nephropathy

IGA Glomerulonephritis
National Center for Research Resources (NCRR)
Yale University

Completed

Phase II Double Blind Placebo Controlled Trial of Risperidone in Tourette Syndrome

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

Completed

Study of Topical Calcitriol in Children With Psoriasis

Psoriasis
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Randomized Study of Cidofovir for Peripheral Cytomegalovirus Retinitis

Acquired Immunodeficiency Syndrome | Cytomegalovirus Retinitis
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone in Women With Active Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
National Institute of Allergy and Infectious Diseases...
National Bacteriological Laboratory

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Acellular and Whole-Cell Pertussis Vaccines

Pertussis
National Center for Research Resources (NCRR)
University of Pennsylvania

Unknown

Diagnostic Study of Quantitative Imaging and Spectroscopy in Patients With Multiple Sclerosis

Multiple Sclerosis

United States
National Center for Research Resources (NCRR)
University of Vermont

Completed

Phase II Placebo-Controlled Study of 4-Aminosalicylic Acid for Ulcerative Colitis

Ulcerative Colitis
National Center for Research Resources (NCRR)
University of Michigan

Completed

Pilot Study of the Effect of Baclofen and Bromocriptine on Luteinizing Hormone Secretion in Pubertal Children

Growth Disorders
National Center for Research Resources (NCRR)
University of Rochester

Completed

Phase II Pilot Controlled Study of Short Vs Longer Term Pimozide (Orap) Therapy in Tourette Syndrome

Tourette Syndrome
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase II Study of the Pathophysiology and Treatment With Enalapril and Polystyrene Sulfonate for Pseudohypoaldosteronism, Type I

Pseudohypoaldosteronism
National Center for Research Resources (NCRR)
University of Pittsburgh

Completed

Pilot Study of Vestibular Rehabilitation Training for Panic Disorder With Vestibular Dysfunction

Vestibular Diseases | Panic Disorder | Agoraphobia
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria

Porphyria
National Center for Research Resources (NCRR)
University of Pennsylvania

Completed

Phase I Study of the Third Generation Adenovirus H5.001CBCFTR in Patients With Cystic Fibrosis

Cystic Fibrosis
National Center for Research Resources (NCRR)
University of Vermont

Completed

Studies of Hereditary Hemorrhagic Telangiectasia

Telangiectasia, Hereditary Hemorrhagic
National Center for Research Resources (NCRR)
University of Pennsylvania

Completed

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

Familial Hypercholesterolemia
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria

Porphyria
National Center for Research Resources (NCRR)
University of Vermont

Completed

Phase III Multicenter Double Blind Controlled Trial of Human Immune Globulin Therapy in Previously Untreated Patients With Chronic Inflammatory Demyelinating Neuropathy

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

Completed

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

Polycystic Ovary Syndrome | Hyperinsulinism
National Center for Research Resources (NCRR)
University of Iowa

Completed

Phase III Randomized, Double-Blind Study of Dexamethasone Vs Dexamethasone/Methylprednisolone Vs Placebo for Bronchopulmonary Dysplasia

Bronchopulmonary Dysplasia
National Center for Research Resources (NCRR)
University of Maryland

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Copolymer 1 for Relapsing-Remitting Multiple Sclerosis

Multiple Sclerosis
National Center for Research Resources (NCRR)
University of Rochester

Completed

Phase II Pilot Study of Olsalazine for Ankylosing Spondylitis

Ankylosing Spondylitis
National Center for Research Resources (NCRR)
University of Michigan

Completed

Phase II Pilot Study of Cytarabine for Refractory Systemic Lupus Erythematosus

Lupus Erythematosus, Systemic
National Center for Research Resources (NCRR)
University of Michigan

Completed

Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Tyrosinemia, Type I
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria

Porphyria
National Institute on Drug Abuse (NIDA)
University of Texas

Completed

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

Panic Disorder | Anxiety Disorder

United States
National Center for Research Resources (NCRR)
University of Tennessee

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Mexiletine for Painful Diabetic Neuropathy

Pain | Diabetic Neuropathies | Paresthesia
National Center for Research Resources (NCRR)
University of Texas

Completed

Study of Nutritional Factors in Porphyria

Porphyria
National Center for Research Resources (NCRR)
University of Rochester

Completed

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Mitochondrial Myopathy
National Center for Research Resources (NCRR)
University of Vermont

Completed

Study of the Natural History and Genotype-Phenotype Correlations of Hereditary Hemorrhagic Telangiectasia Patients

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

Completed

Study of the Effect of Growth Hormone-Releasing Hormone Antagonist on Growth Hormone Release in Acromegaly

Acromegaly
National Center for Research Resources (NCRR)
University of Pittsburgh

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's Phenomenon Secondary to Systemic Sclerosis

Systemic Sclerosis | Raynaud Disease
National Center for Research Resources (NCRR)
University of Texas

Completed

Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria

Phenylketonuria
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Determining Disease Activity Biomarkers in Individuals With Giant Cell Arteritis

Temporal Arteritis

United States, Canada
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

Polyarteritis Nodosa

United States, Canada
National Institute of Allergy and Infectious Diseases...
Istituto Superiore di Sanità

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Acellular and Whole-Cell Pertussis Vaccines

Pertussis
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Recruiting

One-Time DNA Study for Vasculitis

Giant Cell Arteritis | Microscopic Polyangiitis | Polyarteritis Nodosa | Takayasu's Arteritis | Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss) | Granulomatosis With Polyangiitis (Wegener's)

United States, Canada, Turkey
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Abatacept in Treating Adults With Mild Relapsing Wegener's Granulomatosis

Wegener's Granulomatosis

United States
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Longitudinal Protocol for Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis

Microscopic Polyangiitis | Granulomatosis With Polyangiitis | Wegener's

United States, Canada
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Determining Disease Activity Biomarkers in Individuals With Takayasu's Arteritis

Takayasu's Arteritis

United States, Canada
National Center for Research Resources (NCRR)
Tufts Medical Center

Completed

Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis

Liver Cirrhosis, Biliary
National Center for Research Resources (NCRR)
Ohio State University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders

Paralysis, Hyperkalemic Periodic | Paramyotonia Congenita | Hypokalemic Periodic Paralysis
National Center for Research Resources (NCRR)
Penn State University

Completed

Phase II Pilot Study of Early Cortisol Replacement to Prevent Bronchopulmonary Dysplasia

Bronchopulmonary Dysplasia
National Center for Research Resources (NCRR)
Shirley Ryan AbilityLab

Completed

Pilot Study of Fructose for Sickle Cell Crisis

Sickle Cell Anemia

Clinical Trials Sponsored by Office of Rare Diseases (ORD)

Sponsors and Collaborators

Medical Conditions

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CROs in South Africa

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Rare Diseases

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