Clinical Trials Sponsored by Orphan Therapeutics

Total 6690 results

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

Taste and Palatability of Orfadin Suspension

Hereditary Tyrosinemia, Type I

Germany, United Kingdom, France
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

Completed

Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Infant Formula for Preterm Infants

Replacement Therapy in Preterm Infants

Italy
Swedish Orphan Biovitrum

Completed

Characterization of Cyanocobalamin in Healthy Voluteers After Intranasal and Intramuscual Administration

Focus: Bioavailability

Sweden
PTC Therapeutics
Cystic Fibrosis Foundation; FDA Office of Orphan Products Development

Completed

PTC124 for Cystic Fibrosis

Cystic Fibrosis

United States
AOP Orphan Pharmaceuticals AG

Not yet recruiting

Investigational Trial to Evaluate Safety and Tolerability of Treprostinil in Children Diagnosed With PAH

Pulmonary Arterial Hypertension

Spain, Austria, France, Hungary
Amgen

Completed

Evaluating the Effect of Anakinra (r-metHuIL-1ra) on Vaccine AntibodyResponse in Subjects With Rheumatoid Arthritis (RA)

Rheumatoid Arthritis
AOP Orphan Pharmaceuticals AG

Completed

A Short Term Pharmacokinetic, Pharmacodynamic and Tolerability Study to Compare AOP200704 vs. Esmolol

Healthy Volunteers | Pharmacokinetics/Dynamics Study

Czech Republic
Amgen

Terminated

The SPECTRA Study

Rheumatoid Arthritis
Amgen

Completed

Juvenile Rheumatoid Arthritis

Juvenile Chronic Arthritis
Swedish Orphan Biovitrum
Parexel

Completed

A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy Volunteers

Drug Drug Interaction

Germany
Swedish Orphan Biovitrum
Amgen

Completed

Study to Evaluate Palifermin in the Reduction of Dysphagia in Patients With Locally Advanced Non-Small Cell Lung Cancer (NSCLC)

Non-Small Cell Lung Cancer | Lung Cancer | Dysphagia
Swedish Orphan Biovitrum
Amgen

Completed

Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer

Head and Neck Cancer | Mucositis | Stomatitis | Solid Tumors | Squamous Cell Carcinoma
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
AOP Orphan Pharmaceuticals AG

Completed

A Pharmacokinetic, Pharmacodynamic, Safety Study With AOP LDLA202, ONO LDL50 and Esmolol in Healthy Volunteers

Healthy

Czech Republic
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
AOP Orphan Pharmaceuticals AG

Completed

Lancovutide (Moli1901) Inhalation Solution Study in Adolescents and Adults With Cystic Fibrosis

Cystic Fibrosis

Austria
AOP Orphan Pharmaceuticals AG

Enrolling by invitation

A Prospective Non-interventional Observational Study to Observe Long-term Treatment and Outcomes in Pulmonary Arterial Hypertension (PAH) Patients (TripleTRE-NIS)

Pulmonary Arterial Hypertension

France, Spain, Germany, Italy, Hungary, Austria, Czechia, Poland, Romania
AOP Orphan Pharmaceuticals AG

Completed

Safety Study of Pegylated Interferon Alpha 2b to Treat Polycythemia Vera (PEGINVERA)

Polycythemia Vera

Austria
Swedish Orphan Biovitrum
Amgen

Completed

A Study of Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy and Concurrent Chemotherapy

Head and Neck Cancer | Mucositis
AOP Orphan Pharmaceuticals AG

Completed

Anagrelide Retard vs. Placebo: Efficacy and Safety in "At-risk" Patients With Essential Thrombocythaemia (ARETA)

Essential Thrombocythaemia

Poland, Slovakia, Bulgaria, Russian Federation, Romania, Croatia, Lithuania, Austria, Ukraine
AOP Orphan Pharmaceuticals AG

Completed

Anagrelide Retard in Essential Thrombocythemia (TEAM-ET)

Essential Thrombocythemia

Austria, Bulgaria, Lithuania, Poland, Russian Federation
AOP Orphan Pharmaceuticals AG

Completed

Long-term PK and Safety/Tolerability Testing LDLL600 Against Esmolol in Healthy Volunteers

Healthy

Czech Republic
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
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
Swedish Orphan Biovitrum
Amgen

Withdrawn

A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy

Head and Neck Cancer
Orpha Labs

Unknown

Study of ORL-1M (D-mannose) in Patients With CDG-Ib

CDG Ib

Turkey
Orpha Labs

Unknown

Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14

Glycogen Storage Disease, Type 14

Turkey
Orpha Labs

Completed

Study of ORL-1B in Patients With Biotinidase Deficiency

Biotinidase Deficiency
Steven Hays, MD
OrphAI Therapeutics

Recruiting

LAM-001 in Lung Transplant Recipients With Bronchiolitis Obliterans Syndrome. (INSPO-BOS)

Lung Transplantation | Chronic Lung Allograft Dysfunction | Bronchiolitis Obliterans Syndrome

United States
Swedish Orphan Biovitrum
Cerner Enviza

Completed

Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination (A-MOVE)

Hemophilia A

France
Swedish Orphan Biovitrum
Cerner Enviza

Completed

rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

Hemophilia A With Inhibitor

France, Ireland, Italy, Norway, Germany, Kuwait, Saudi Arabia, Switzerland
Vicore Pharma AB
Orphan Reach

Completed

Safety and Efficacy of C21 in Subjects With COVID-19

COVID-19

India, United Kingdom
Orpha Labs

Completed

Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II

Leukocyte Adhesion Deficiency, Type II
Northwell Health
Swedish Orphan Biovitrum

Recruiting

Effects of Anakinra in Subjects With Corticosteroid-resistant/Intolerant Meniere's Disease and Autoimmune Inner Ear Disease

Meniere's Disease | Autoimmune Inner Ear Disease

United States
Swedish Orphan Biovitrum
Syneos Health

Active, not recruiting

A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (FREEDOM)

Hemophilia A, Severe

Germany, Spain, France, Czechia, Netherlands, Ireland, Austria, Croatia, Greece, Italy, Norway, Slovenia, Sweden, United Kingdom
Orphan Medical
Johns Hopkins University

Completed

Physiological, Behavioral and Subjective Effects of Drugs (GHB)

Sedative Abuse

United States
Vicore Pharma AB
Orphan Reach

Completed

Safety, Efficacy and Pharmacokinetics of C21 in Subjects With IPF

Idiopathic Pulmonary Fibrosis

United Kingdom, India, Ukraine, Russian Federation
Swedish Orphan Biovitrum
Cerner Enviza

Active, not recruiting

An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B (B-MORE)

Hemophilia B

Czechia, Greece, Ireland, Italy, Norway, Saudi Arabia, Spain, Sweden, United Kingdom
Swedish Orphan Biovitrum
Cerner Enviza

Completed

A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France (B-SURE)

Haemophilia B

France
Swedish Orphan Biovitrum
ICON plc

Completed

Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products

Hemophilia A

France, Italy, Norway, Sweden, United Kingdom, Austria, Belgium, Finland, Spain
Skane University Hospital
Indiana Hemophilia &Thrombosis Center, Inc.; Bioverativ Therapeutics Inc.; Swedish...

Active, not recruiting

Natural History Study of Factor IX Treatment and Complications (B-Natural)

Factor IX Deficiency

United States
Swedish Orphan Biovitrum
Apellis Pharmaceuticals, Inc.

Recruiting

Evaluate the PK Efficacy Safety and Tolerability of Pegcetacoplan in Patients With Thrombotic Microangiopathy

Transplant-Associated Thrombotic Microangiopathy

United States, France, Greece, Italy, Spain
University Hospital, Bonn
Swedish Orphan Biovitrum

Recruiting

Sonography and Haemophilia

Haemophilia | Sonography

Germany
FDA Office of Orphan Products Development

Completed

An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors

Neurofibromatosis 2

United States
FDA Office of Orphan Products Development

Completed

Risk-adapted Therapy for Primary Systemic (AL) Amyloidosis

Amyloidosis
FDA Office of Orphan Products Development

Completed

Keratinocyte Growth Factor to Prevent Acute GVHD

Graft-vs-Host Disease

United States

Clinical Trials Sponsored by Orphan Therapeutics

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Equatorial Guinea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations