Clinical Trials Sponsored by Orphan Therapeutics

Total 6594 results

OrphAI Therapeutics

Recruiting

A Phase 2a Study of LAM-001 for the Treatment of Pulmonary Hypertension

Pulmonary Hypertension

United States
Swedish Orphan Biovitrum
Bioverativ Therapeutics Inc.

Completed

Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies (ReITIrate)

Hemophilia A

Ireland, United Kingdom, Germany, United States, Canada, Slovenia, Sweden
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A

Severe Hemophilia A

United States, United Kingdom, Spain, Hong Kong, Israel, New Zealand, Sweden, Australia, Brazil, Japan, Austria, Belgium, Italy, India, Switzerland, Canada, France, Germany, South Africa
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale (Elevate)

Severe Hemophilia A

United States, Australia, New Zealand
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A (Kids ALONG)

Hemophilia A

United States, Poland, United Kingdom, Netherlands, South Africa, Hong Kong, Australia, Ireland
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

Hemophilia B

United States, United Kingdom, South Africa, Netherlands, Ireland, Australia
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Terminated

Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes (FORWARD)

Hemophilia A | Hemophilia B

United States
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

Severe Hemophilia B

Sweden, United States, France, Italy, Russian Federation, United Kingdom, Germany, China, Poland, Japan, Australia, Brazil, Canada, India, South Africa, Hong Kong, Belgium
Point Therapeutics

Completed

Study of Talabostat and Rituximab in Advanced Chronic Lymphocytic Leukemia (CLL)

Chronic Lymphocytic Leukemia

United States
United Therapeutics

Completed

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

Pulmonary Hypertension

United States
Orphan Australia

Completed

VSL#3 Versus Placebo in Maintenance of Remission in Crohn's Disease

Crohn's Disease

Australia
Jupiter Orphan Therapeutics Inc.
National Institute on Aging (NIA)

Completed

JOTROL PK, Safety, and Food Effect Assessment

Pharmacokinetics | Food-drug Interaction | Safety

United States
Orphan Medical

Completed

Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients

Narcolepsy

United States, Canada, Switzerland
Swedish Orphan Biovitrum

Suspended

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

Cold Agglutinin Disease

Spain, Belgium, Norway, United States, Japan, United Kingdom, Netherlands, Canada, Hungary, Austria, Finland, Georgia, Germany, Italy
Swedish Orphan Biovitrum

Active, not recruiting

A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients

Primary Hemophagocytic Lymphohistiocytosis

China
Swedish Orphan Biovitrum

Active, not recruiting

An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP (ADOPT)

Immune Thrombocytopenia | ITP

Croatia, Czechia, Germany, Italy, Netherlands, Norway, Spain, Switzerland, United Kingdom
Swedish Orphan Biovitrum

Completed

A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.

Rare Diseases

Japan
Swedish Orphan Biovitrum

Completed

Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.

Healthy

Netherlands
Swedish Orphan Biovitrum

Terminated

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD) (anaSTILLs)

Still's Disease, Adult-Onset | Still's Disease, Juvenile-Onset

United States, Canada
Swedish Orphan Biovitrum

Completed

Kineret in the Treatment of Rheumatoid Arthritis

Rheumatoid Arthritis

Germany
Swedish Orphan Biovitrum

Completed

A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia Care

Haemophilia A | Haemophilia B

France, Germany, Italy, Spain, Sweden, Belgium, United Kingdom
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Sweden
Swedish Orphan Biovitrum

Terminated

Comparison of rhBSSL With Placebo When Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm Infants (LAIF)

Prevention of Growth Restriction

Belgium, France, Germany, Hungary, Italy, Poland, Spain, Sweden, Czech Republic, Russian Federation
Swedish Orphan Biovitrum

Completed

A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma

Multiple Myeloma

Germany
Swedish Orphan Biovitrum

Completed

Palifermin DDI (Drug Drug Interaction)

Healthy Volunteers
Swedish Orphan Biovitrum

Completed

PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation

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

Recruiting

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Hereditary Tyrosinemia, Type I

China
Swedish Orphan Biovitrum

Recruiting

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE (EMERALD)

Systemic Lupus Erythematosus | Macrophage Activation Syndrome | Still Disease | Secondary Hemophagocytic Lymphohistiocytosis | SJIA | AOSD | MAS

United States, Belgium, Italy, Netherlands, United Kingdom, China, France, Spain, Czechia, Canada, Sweden, Japan, Germany, Poland
Swedish Orphan Biovitrum

Active, not recruiting

A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health (A-MORE)

Hemophilia A

Spain, Germany, Czechia, Italy, Estonia, Finland, Greece, Netherlands, Saudi Arabia, Slovenia, Sweden, Switzerland, United Kingdom
Swedish Orphan Biovitrum

Completed

Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis

Primary Hemophagocytic Lymphohistiocytosis

Spain, United States, United Kingdom, Germany, Italy, Sweden, Canada, Switzerland
Swedish Orphan Biovitrum

Completed

Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B (MIND)

Haemophilia A | Haemophilia B

Sweden, Finland, Denmark
Swedish Orphan Biovitrum

Completed

First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy Volunteers

Healthy Volunteers

United Kingdom
Swedish Orphan Biovitrum

Completed

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

Haemophilia A | Haemophilia B

Germany
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Turkey
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Germany, Turkey
Swedish Orphan Biovitrum

Completed

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis (anaGO)

Acute Gouty Arthritis

United States
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Swedish Orphan Biovitrum

Completed

Long-term Safety Study of Kineret® in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)

Still Disease, Juvenile Onset

Italy
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

Study to Characterize the Effect of Heparin on Palifermin Activity

Oral Mucositis

United States
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Pasteurized Breast Milk for Preterm Infants

Replacement Therapy in Preterm Infants

France, Italy
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum; Syntonix Pharmaceuticals, Inc.

Completed

Phase I/IIa Study of FIXFc in Hemophilia B Patients

Hemophilia B

United States
Swedish Orphan Biovitrum

Recruiting

A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (COMPLETE)

Paroxysmal Nocturnal Hemoglobinuria

Spain
Swedish Orphan Biovitrum

Completed

A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy (DISSOLVE I)

Chronic Gout

United States
Swedish Orphan Biovitrum

Completed

A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy II (DISSOLVE II)

Chronic Gout

United States, Serbia, Georgia, Ukraine, Russian Federation
Swedish Orphan Biovitrum

Completed

A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)

Arthritis, Juvenile | Lymphohistiocytosis, Hemophagocytic | Macrophage Activation Syndrome | Adult Onset Still Disease

United States, Spain, United Kingdom, France, Italy
Swedish Orphan Biovitrum

Terminated

Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patients With COVID-19 Infection.

SARS-CoV-2

United States, Italy
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

Clinical Trials Sponsored by Orphan Therapeutics

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mauritius

Conditions

Rare Diseases

Drug Interventions

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