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
Large B-cell Lymphoma, Relapsed Non-Hodgkin Lymphoma
Emapalumab Prevention of CAR-T Cell Associated Toxicities
This research study involves assessing the impact of emapalumab as preventative management of CAR-T related cytokine release syndrome in participants with Non-Hodgkin's lymphoma (NHL).
- Conditions
- Large B-cell Lymphoma, Relapsed Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma
- Trial name
- NCT06550141
- Testing
- Emapalumab, Cyclophosphamide, Fludarabine Phosphate
- Where
- 2 sites in United States
- Volunteers
- 28 people
- Sponsor
- Marcela V. Maus, M.D.,Ph.D.
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
Myelofibrosis，MF
Pacritinib For Bone Marrow Fibrosis In Patients With Myelofibrosis Who Have Thrombocytopenia
We hypothesize that pacritinib leads to modification of the myelofibrosis (MF) disease phenotype, especially related to BM fibrosis and cytopenias; due potentially to its dual effect as an inhibitor of the JAK and NFκB pathways…
- Trial name
- PACRIMYEL (NCT07394153)
- Testing
- Pacritinib
- Where
- 13 sites in Spain
- Volunteers
- 30 people
- Sponsor
- Grupo Español de Enfermedades Mieloproliferativas Crónicas PH Negativas
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
Gastrointestinal Cancer, Gastrointestinal Neoplasms
Avatrombopag vs. Placebo for CIT in GI Malignancies
The purpose of this study is to compare the efficacy of two study drugs, Avatrobopag versus placebo, to treat persistent Chemotherapy-Induced Thrombocytopenia (CIT) in patients with gastrointestinal (GI) malignancies receiving…
- Conditions
- Gastrointestinal Cancer, Gastrointestinal Neoplasms, Chemotherapy-Induced Thrombocytopenia
- Trial name
- ACT-GI (NCT05772546)
- Testing
- Avatrombopag
- Where
- 4 sites in United States
- Volunteers
- 49 people
- Sponsor
- Hanny Al-Samkari, MD
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
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
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
Dermatomyositis, Dermatomyositis Sine Myositis
Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study
This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial lung disease (MDA5 RP-ILD).
- Conditions
- Dermatomyositis, Dermatomyositis Sine Myositis, Dermatomyositis With Myopathy, Dermatomyositis With Respiratory Involvement
- Trial name
- NCT07486869
- Testing
- Emapalumab
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Miami
Accelerated Phase Myeloproliferative Neoplasm, Blast Phase Myeloproliferative Neoplasm
Pacritinib With Standard of Care Azacitidine or Decitabine as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant for Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms
This phase II trial tests if adding pacritinib to standard of care azacitidine or decitabine increases the number of patients able to proceed to hematopoietic stem cell transplantation (bridging) for patients with accelerated and…
- Trial name
- NCT07148947
- Testing
- Pacritinib, Decitabine, Decitabine and Cedazuridine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Washington
Mild Hemophilia
Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%)
Currently, few recommendations exist for patients with mild hemophilia, who represent approximately 60% of the hemophilia population.
- Trial name
- echoSAHM (NCT07719647)
- Testing
- Joint Ultrasound (HEAD-US), Blood Sample for Coagulation Factor Measurement
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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