Clinical trials: sponsored by Swedish Orphan Biovitrum
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
This study aims to evaluate safety information in patients with HLH/MAS in Still's disease exposed to Gamifant.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS), Still Disease
- Trial name
- NCT07909005
- Testing
- Local laboratory testing, Local Lab Tests
- Where
- 18 sites in United States
- Volunteers
- 70 people
- Sponsor
- Swedish Orphan Biovitrum
C3 Glomerulopathy (C3G), Primary Immune-Complex Membranoproliferative
To Assess the Efficacy and Safety of Pegcetacoplan for the Prevention of Recurrent C3G or Primary IC-MPGN in Kidney Transplant Recipients
To assess the efficacy and safety of pegcetacoplan for the prevention of recurrent C3G or primary IC-MPGN in kidney transplant recipients
- Conditions
- C3 Glomerulopathy (C3G), Primary Immune-Complex Membranoproliferative, Glomerulonephritis (IC-MPGN), Kidney Transplantation
- Trial name
- PRESERVE (NCT07898774)
- Testing
- Pegcetacoplan
- Where
- 33 sites in Brazil, Canada and 8 more
- Volunteers
- 30 people
- Sponsor
- Swedish Orphan Biovitrum
Myelofibrosis (MF)
Pacritinib Effectiveness in Real-world Settings
This study aims to evaluate real-world treatment patterns and effectiveness of pacritinib, including hematologic and clinical outcomes, and survival through a site-based retrospective chart review of medical records of patients…
- Trial name
- PACER (NCT07774455)
- Testing
- Not applicable- observational study
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Swedish Orphan Biovitrum
Haemophilia A (Moderate or Severe)
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study…
- Trial name
- LIBERTY (NCT06716814)
- Testing
- Efanesoctocog alfa
- Where
- 20 sites in Bulgaria, France and 5 more
- Volunteers
- 89 people
- Sponsor
- Swedish Orphan Biovitrum
VEXAS, VEXAS Syndrome
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome.
- Trial name
- NCT06782373
- Testing
- Pacritinib
- Where
- 39 sites in United States, Canada and 6 more
- Volunteers
- 156 people
- Sponsor
- Swedish Orphan Biovitrum
Macrophage Activation Syndrome, Still's Disease, Adult-Onset
Assessment of Macrophage Activation syndromE in STill's Disease
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
- Conditions
- Macrophage Activation Syndrome, Still's Disease, Adult-Onset, Stills Disease, Juvenile-Onset
- Trial name
- AMETHYST (NCT06405152)
- Testing
- Chart review
- Where
- 11 sites in United States, Canada and 5 more
- Volunteers
- 75 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a…
- Trial name
- A-MORE (NCT04293523)
- Testing
- ELOCTA
- Where
- 53 sites in Czechia, Estonia and 12 more
- Volunteers
- 427 people
- Sponsor
- Swedish Orphan Biovitrum
C3 Glomerulopathy (C3G), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study
The purpose of this study is to evaluate the effectiveness and safety of Pegcetacoplan in patients with C3G and primary IC-MPGN in the real-world setting.
- Trial name
- NCT07746895
- Testing
- Pegcetacoplan
- Where
- 101 sites in Australia, Austria and 12 more
- Volunteers
- 150 people
- Sponsor
- Swedish Orphan Biovitrum
Paroxysmal Nocturnal Hemoglobinuria
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH.
- Trial name
- COMPLETE (NCT05776472)
- Testing
- Pegcetacoplan
- Where
- 87 sites in Australia, Belgium and 13 more
- Volunteers
- 165 people
- Sponsor
- Swedish Orphan Biovitrum
Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis
Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic juvenile idiopathic arthritis…
- Conditions
- Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus
- Trial name
- EMERALD (NCT05001737)
- Testing
- Emapalumab
- Where
- 44 sites in United States, Belgium and 11 more
- Volunteers
- 33 people
- Sponsor
- Swedish Orphan Biovitrum
Primary Hemophagocytic Lymphohistiocytosis
A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.
- Trial name
- NCT05744063
- Testing
- Emapalumab-Lzsg 5 MG/ML [Gamifant]
- Where
- 7 sites in China
- Volunteers
- 13 people
- Sponsor
- Swedish Orphan Biovitrum
Aplastic Anemia
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment
This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells.
- Trial name
- NCT07786922
- Testing
- Avatrombopag 20 mg Oral Tablet
- Volunteers
- 26 people
- Sponsor
- Swedish Orphan Biovitrum
Systemic Juvenile Idiopathic Arthritis (sJIA), Adult Onset Still's Disease (AOSD)
An Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD)
The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD).The study consists of up to four weeks…
- Trial name
- NCT07706868
- Testing
- Anakinra (Kineret®)
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Severe
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis.
- Trial name
- FREEDOM (NCT05817812)
- Testing
- Efanesoctocog alfa
- Where
- 30 sites in Austria, Croatia and 12 more
- Volunteers
- 93 people
- Sponsor
- Swedish Orphan Biovitrum
Familial Mediterranean Fever (FMF )
A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF
The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF).
- Trial name
- NCT06666335
- Testing
- Anakinra
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
Sobi.BIVV001-003 is an open-label, 2-period, fixed sequence study for intra-participant comparison of the PK profiles of efanesoctocog alfa and the extended half-life rFVIII products damactocog alfa pegol or turoctocog alfa pegol…
- Trial name
- NCT06579144
- Testing
- Efanesoctocog alfa
- Where
- 7 sites in Bulgaria, Germany and 2 more
- Volunteers
- 24 people
- Sponsor
- Swedish Orphan Biovitrum
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count <50,000/μL).
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- PACIFICA (NCT03165734)
- Testing
- Pacritinib, Physician's Choice medications
- Where
- 207 sites in United States, Australia and 22 more
- Volunteers
- 407 people
- Sponsor
- Swedish Orphan Biovitrum
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum