Search clinical trials for: orphan disease

Total 383677 results

The Cleveland Clinic

Terminated

GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Pulmonary Alveolar Proteinosis

United States
M.D. Anderson Cancer Center

Completed

Clofarabine in Chronic Lymphocytic Leukemia

Lymphoproliferative Disorders | Leukemia | Hematologic Neoplasms | Leukemia, Lymphocytic, Chronic

United States
University of Florida

Completed

Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis

Lactic Acidosis
Cellegy Pharmaceuticals

Completed

Phase III Study of Monolaurin Cream Therapy for Patients With Congenital Ichthyosis

Ichthyosis
FDA Office of Orphan Products Development
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Completed

Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

Adrenoleukodystrophy
Icahn School of Medicine at Mount Sinai

Completed

Randomized Study of Fluoxetine in Children and Adolescents With Autism

Autism

United States
University of Michigan

Completed

Sincalide (Cholecystokinin Octapeptide) Versus Placebo in Neonates at High Risk for Developing Parenteral Nutrition Associated Cholestasis

Cholestasis

United States
FDA Office of Orphan Products Development
SciClone Pharmaceuticals

Completed

Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis

Cystic Fibrosis
Children's Hospital Medical Center, Cincinnati

Completed

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Osteopenia | Gaucher's Disease

Israel, United States
FDA Office of Orphan Products Development

Completed

Minocycline in Patients With Huntington's Disease

Huntington's Disease

United States
Beth Israel Deaconess Medical Center

Completed

Study of Recombinant Human Insulin-Like Growth Factor I in Patients With Severe Insulin Resistance

Hyperglycemia | Insulin Resistance

United States
University of North Carolina

Completed

Randomized Study of Alendronate in Adult Patients With Cystic Fibrosis Related Osteoporosis

Osteoporosis | Cystic Fibrosis

United States
United Therapeutics

Completed

Phase III Randomized Study of UT-15 in Patients With Primary Pulmonary Hypertension

Pulmonary Hypertension

United States
Icahn School of Medicine at Mount Sinai

Completed

Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable Immunodeficiency

Common Variable Immunodeficiency
Seattle Children's Hospital

Completed

Study of Morphine in Postoperative Infants to Allow Normal Ventilation

Pain | Infant, Newborn, Diseases
Massachusetts General Hospital

Completed

Androgen Replacement Therapy in Women With Hypopituitarism

Hypopituitarism

United States
University of Pennsylvania

Terminated

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

Ornithine Transcarbamylase Deficiency Disease
Orfagen
FDA Office of Orphan Products Development

Completed

Systemic and Local Diffusion of Ethanol After Administration of Ethanol 96% Formulated in a Gel and Ethanol 98% Solution by the Percutaneous Route, in Patients With Congenital Venous Malformations:Pharmacokinetic, Pharmacodynamic and Clinical Study.

Congenital Venous Malformation

United States, France
Swedish Orphan Biovitrum

Completed

A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCT

Leukemia

United States
Swedish Orphan Biovitrum

Completed

Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency

Cystic Fibrosis | Exocrine Pancreatic Insufficiency

Netherlands, Poland
Swedish Orphan Biovitrum

Completed

Study to Characterize the Effect of Heparin on Palifermin Activity

Oral Mucositis

United States
Swedish Orphan Biovitrum

Completed

Taste and Palatability of Orfadin Suspension

Hereditary Tyrosinemia, Type I

Germany, United Kingdom, France
Swedish Orphan Biovitrum

Completed

PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation

Multiple Myeloma | Non-Hodgkin's Lymphoma
Swedish Orphan Biovitrum

Completed

Efficacy and Tolerability of Novel A2A Agonist in Treatment of Diabetic Neuropathic Pain

Diabetic Neuropathic Pain

Czech Republic, Germany, South Africa
Swedish Orphan Biovitrum
Amgen

Completed

Pharmacokinetic Study of Palifermin in Subjects Receiving Radiation Therapy and Chemotherapy Followed by Blood Stem Cell Support

Lymphoma | Leukemia | Myeloma
Swedish Orphan Biovitrum
Amgen

Completed

Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)

Lymphoma | Leukemia | Cancer
Swedish Orphan Biovitrum
Amgen

Completed

Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Allogeneic Marrow/Peripheral Blood Progenitor Cell (PBPC) Transplantation

Hematologic Malignancies | Graft Versus Host Disease
Pastoral Activities and Service for AIDS Dar-Es-Salaam...
King's College London; University of Cape Town

Unknown

Phase II Randomised Controlled Trial of Memory Work Therapy at PASADA, Tanzania (MWT)

HIV

Tanzania
University of Edinburgh
Medical Research Council; NHS Lothian

Unknown

Heme Arginate in Cardiac Surgery Patients (HACS)

Acute Kidney Injury

United Kingdom
Swedish Orphan Biovitrum

Completed

Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension

Healthy Volunteers

Netherlands
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Orphan...

Completed

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

Hyperoxaluria

United States
University of Tennessee
FDA Office of Orphan Products Development

Completed

Radioimmunoimaging of AL Amyloidosis

Primary Amyloidosis

United States
UCSF Benioff Children's Hospital Oakland
Department of Health and Human Services; FDA Office of Orphan Products Development

Completed

Simvastatin (Zocor) Therapy in Sickle Cell Disease

Sickle Cell Disease

United States
Merit Cudkowicz
FDA Office of Orphan Products Development

Completed

Pilot Study of Minocycline in Huntington's Disease

Huntington Disease

United States, Canada
Patricia I. Dickson, M.D.
University of California, Los Angeles; FDA Office of Orphan Products Development and other collaborators

Terminated

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Mucopolysaccharidosis I | Lysosomal Storage Diseases | Spinal Cord Compression

United States, Finland
University of Arizona
FDA Office of Orphan Products Development

Terminated

Safety and PK of Nikkomycin Z for Coccidioides Pneumonia Treatment

Coccidioidomycosis

United States
ISDIN
Digna Biotech S.L.

Completed

Efficacy and Safety Study of p144 to Treat Skin Fibrosis in Systemic Sclerosis

Skin Fibrosis

Spain, United Kingdom, Germany, Poland, Hungary, Italy
Bioprojet

Completed

Efficacy and Safety Study of BF2.649 and BF2.649 Add on Modafinil on Cataplexy in Patients With Narcolespy (Harmony2)

Narcolepsy | Cataplexy | Excessive Daytime Sleepiness

Switzerland
ISDIN
Digna Biotech S.L.

Completed

Open Label Extension (OLE) for the Patients Treated in the ISD002-P144-07 Study

Skin Fibrosis

United Kingdom, Spain, Italy, Poland, Germany, Hungary
Penn State University

Withdrawn

C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation

Hereditary Angioedema
Bioprojet

Completed

Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy (Harmony1)

Sleep Disorders | Narcolepsy | Cataplexy | Excessive Daytime Sleepiness

Switzerland
Emphycorp
Cellular Sciences, inc.

Completed

Inhaled Sodium Pyruvate for the Treatment of Cystic Fibrosis.

Cystic Fibrosis

United States
Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development

Completed

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

Fanconi Anemia

United States
Genethon

Completed

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Limb Girdle Muscular Dystrophy Type 2C | Gamma-sarcoglycanopathy

France
Medical University of South Carolina
Merck Sharp & Dohme LLC; FDA Office of Orphan Products Development

Completed

Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis

Osteoporosis

United States
University of Florence

Completed

High-dose Ibuprofen for Patent Ductus Arteriosus (PDA) in Preterm Infant

Ductus Arteriosus, Patent

Italy
AOP Orphan Pharmaceuticals AG

Completed

Anagrelide vs. Hydroxyurea - Efficacy and Tolerability Study in Patients With Essential Thrombocythaemia (ANAHYDRET)

Essential Thrombocythaemia

Austria, Czech Republic, France, Germany, Hungary, Italy, Lithuania, Poland, Singapore, Slovenia
Institut National de la Santé Et de la Recherche...

Completed

Interleukin-1 Receptor Antagonist (IL-1RA) (ANAKINRA) IN SEVERE SYSTEMIC-ONSET JUVENILE IDIOPATHIC ARTHRITIS

Systemic-Onset Juvenile Idiopathic Arthritis

France
Amgen

Completed

Evaluating Kineret® (Anakinra) in Rheumatoid Arthritis (RA) Subjects Using aSelf-Reported Questionnaire

Rheumatoid Arthritis
Amgen

Completed

Evaluation of the Efficacy of Combination Treatment With Anakinra and Pegsunercept in Improving Rheumatoid Arthritis

Rheumatoid Arthritis

Search clinical trials for: orphan disease

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