Clinical trials: sponsored by Swedish Orphan Biovitrum
Mucopolysaccharidosis III
Open-label Study of Anakinra in MPS III
Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a disorder of metabolism, associated with insufficient production of a lysosomal enzyme needed for normal cell function.
- Trial name
- NCT04018755
- Testing
- anakinra
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Lynda E Polgreen
Haemophilia A, Haemophilia B
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in…
- Trial name
- PREVENT (NCT03055611)
- Testing
- ELOCTA, ALPROLIX
- Where
- 22 sites in Germany
- Volunteers
- 201 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period.
- Trial name
- NCT02976753
- Testing
- efmoroctocog alfa, Factor VIII
- Where
- 36 sites in Austria, Belgium and 7 more
- Volunteers
- 361 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophagocytic Lymphohistiocytoses
Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis
This observational study is designed to provide a better understanding of the potential use of interferon gamma (IFNγ) and IFNγ-inducible chemokines as markers of HLH disease activity in adults, and the potential of IFNγ to…
- Trial name
- NCT06047210
- Testing
- Blood Draws Data Collection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Swedish Orphan Biovitrum
Hereditary Tyrosinemia, Type I
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
The purpose of this study is to look at the long term safety profile of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1 (HT-1).
- Trial name
- OPAL (NCT02320084)
- Testing
- Nitisinone
- Where
- 77 sites in Austria, Belgium and 15 more
- Volunteers
- 315 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Hemophilia B
Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)
The purpose of this study is to validate the newly developed Hemophilia Functional Ability Scoring Tool (Hemo-FAST), which is a fast and simple scoring in haemophilia able to assess patient-reported functional mobility.
- Trial name
- Hemo-FAST (NCT04731701)
- Testing
- Hemophilia Joint Health Score (HJHS)
- Where
- 16 sites in France
- Volunteers
- 180 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination
This low interventional study aims to describe if and how the haemophilia treatment management decisions are impacted by a systematic joint examination (ultrasound, functional, physical) in patients with haemophilia A in France.
- Trial name
- A-MOVE (NCT04133883)
- Testing
- Ultrasound, Haemophilia Joint Health Score
- Where
- 20 sites in France
- Volunteers
- 88 people
- Sponsor
- Swedish Orphan Biovitrum
Still Disease, Juvenile Onset
Long-term Safety Study of Kineret® in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)
The purpose of the study is to evaluate and characterize long-term safety of Kineret when used in standard clinical practice to treat patients with systemic juvenile idiopathic arthritis (SJIA).
- Trial name
- NCT03932344
- Testing
- Anakinra
- Where
- Italy
- Volunteers
- 306 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophagocytic Lymphohistiocytosis
Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis
This observational study is designed to determine the levels of pro-inflammatory markers in patients diagnosed with M-HLH, and to assess whether the cytokine profiles bear an IFNγ signature.
- Trial name
- NCT03259230
- Testing
- Blood Draws, Data Collection
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A With Inhibitor
rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
- Trial name
- NCT03951103
- Testing
- rFVIIIFc
- Where
- 18 sites in France, Germany and 6 more
- Volunteers
- 44 people
- Sponsor
- Swedish Orphan Biovitrum
Haemophilia B
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic…
- Trial name
- B-SURE (NCT03655340)
- Testing
- Alprolix
- Where
- 18 sites in France
- Volunteers
- 91 people
- Sponsor
- Swedish Orphan Biovitrum
Haemophilia A, Synovitis
Prevalence of Synovitis in Patients With Haemophilia A
The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.
- Conditions
- Haemophilia A, Synovitis, Hemophilia Arthropathy, Sonography
- Trial name
- SynoPrev (NCT06352216)
- Testing
- Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)
- Where
- Germany
- Volunteers
- 300 people
- Sponsor
- University Hospital, Bonn
Primary Hemophagocytic Lymphohistiocytosis
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term…
- Trial name
- NCT03312751
- Testing
- Emapalumab
- Where
- 27 sites in United States, Canada and 6 more
- Volunteers
- 35 people
- Sponsor
- Swedish Orphan Biovitrum
Haemophilia A, Haemophilia B
Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B
The puropse of this non-interventional register and survey study is to identify the patterns of prescribed pain, anti-depressive and anti-anxiety medication and management of pain, depression and anxiety for people with…
- Trial name
- MIND (NCT03276130)
- Where
- 5 sites in Denmark, Finland and 1 more
- Volunteers
- 2,300 people
- Sponsor
- Swedish Orphan Biovitrum
Kawasaki Disease
A Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment, in Patients With Kawasaki Disease Who Failed to Respond to Initial Standard IVIG Treatment
Kawasaki disease (KD) is the most frequent vasculitis in younger children <5years, and the first cause of acquired ischemic myocardiopathy in childhood.
- Trial name
- ANACOMP (NCT04656184)
- Testing
- ANAKINRA, Intravenous immunoglobulin
- Where
- France
- Volunteers
- 84 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Graft Failure
Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.
This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF.
- Trial name
- NCT04731298
- Testing
- Emapalumab
- Where
- 5 sites in Australia, Canada and 1 more
- Volunteers
- 2 people
- Sponsor
- Swedish Orphan Biovitrum
Rare Diseases
A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects.
- Trial name
- NCT04765553
- Testing
- NI-0501, Saline
- Where
- Japan
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Swedish Orphan Biovitrum
Hemophagocytic Lymphohistiocytoses
A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH
Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on the background of several clinical conditions (e.g.
- Trial name
- NCT03985423
- Testing
- Emapalumab-Lzsg
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Swedish Orphan Biovitrum
Healthy Volunteers
First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy Volunteers
The purpose of this study is to determine the safety, pharmacodynamic and pharmacokinetic profiles of a novel therapeutic drug when administered to healthy volunteers.
- Trial name
- NCT01459562
- Testing
- NI-0501
- Where
- 2 sites in United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Swedish Orphan Biovitrum