Clinical Trials Sponsored by Foundation for Rare Diseases

Total 496 results

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
The Foundation for Spinal Research, Education and...

Unknown

Human Autograft Mesenchymal Stem Cell Mediated Stabilization of The Degenerative Lumbar Spine

Lower Back Pain | Chronic Lower Back Pain | Lumbar Degenerative Disc Disease

United States
Christiana Care Health Services
The Cardeza Foundation for Hematologic Research

Completed

Mechanism of Thrombocytopenia in Infants

Thrombocytopenia | Preeclampsia | Small for Gestational Age (SGA)

United States
Intermountain Health Care, Inc.
Centers for Disease Control and Prevention

Recruiting

Improving Antibiotic Use in Urgent Care Facilities (SCORE_UC)

Antibiotic Side Effect | Behavioral Changes

United States
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Completed

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Thrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
University Research Foundation for Lysosomal Storage...
University of Pittsburgh

Completed

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

Gaucher Disease

United States
Centers for Disease Control and Prevention
Gilead Sciences; CDC Foundation

Completed

Sustainable Healthcenter Implementation PrEP Pilot Study (SHIPP)

HIV Preexposure Prophylaxis | HIV Chemoprophylaxis

United States
Marshfield Clinic Research Foundation
Centers for Disease Control and Prevention

Active, not recruiting

Randomized Influenza Vaccine Evaluation of Immune Response (RIVER)

Influenza

United States
Marshfield Clinic Research Foundation
Centers for Disease Control and Prevention

Completed

Open-Label Influenza Vaccine Evaluation (OLIVE)

Immune Response

United States
Marshfield Clinic Research Foundation
Centers for Disease Control and Prevention

Completed

Efficacy Study of Early Versus Late Oseltamivir Administration for Treating and Preventing Influenza

Influenza

United States
International Centre for Diarrhoeal Disease Research...
UBS Optimus Foundation

Completed

Reduce Childhood Maltreatment and Promote Development

Maltreatment

Bangladesh
University Hospital, Lille
APEHDia, a french CDH patient association; Rare Disease Foundation, France

Not yet recruiting

Engagement in Physical Activities and Sports in Adolescents With Diaphragmatic Hernia (CHASAM)

Congenital Diaphragmatic Hernia
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)
Ohio State University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Intravenous Immune Globulin for Chronic Inflammatory Demyelinating Polyneuropathy

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
Center for Rare Neurological Diseases, Norcross...
Ultragenyx Pharmaceutical Inc; Rett Syndrome Research Trust

Unknown

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

Rett Syndrome

United States
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 Center for Research Resources (NCRR)
Children's Hospital Colorado

Unknown

Phase I Study of Colchicine Therapy in Childhood Hepatic Cirrhosis

Liver Cirrhosis | Cirrhosis
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)
Johns Hopkins University

Completed

Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders

Amino Acid Metabolism, Inborn Errors
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)
Ohio State University

Completed

Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease

Amyotrophic Lateral Sclerosis | Spinal Muscular Atrophy | Spinobulbar Muscular Atrophy
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)
Virginia Commonwealth University

Completed

Study of Individualized Instruction Versus Pamphlet in Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
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
Nantes University Hospital
MAREP ( Center For Rare Diseases Center Ano-Rectal And Pelvic Abnormalities)

Completed

National Study on the Quality of Life of Patients With Anorectal Malformation (MARQOL)

Anorectal Malformation

France
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)
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)
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

Clinical Trials Sponsored by Foundation for Rare Diseases

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