Clinical trials: sponsored by Swedish Orphan Biovitrum
E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin…
- Conditions
- E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS, Vexas Syndrome
- Trial name
- NCT06538181
- Testing
- Pacritinib
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Washington University School of Medicine
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
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
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
Autoimmune Inner Ear Disease, Meniere's Disease
Effects of Anakinra in Subjects With Corticosteroid-resistant/Intolerant Meniere's Disease and Autoimmune Inner Ear Disease
A Phase II randomized, placebo controlled study design of anakinra (Kineret) in corticosteroid-resistant or intolerant Meniere's disease (CR-MD)and corticosteroid-resistant or intolerant autoimmune inner ear disease (CR-AIED)…
- Trial name
- NCT03587701
- Testing
- Anakinra 100Mg/0.67Ml Inj Syringe (Period 1), Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Northwell Health
Leukemia, Multiple Myeloma
Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation
The purpose of this study is to help determine if palifermin and leuprolide acetate can help the immune system recover faster following a stem cell transplant.
- Conditions
- Leukemia, Multiple Myeloma, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma
- Trial name
- NCT01746849
- Testing
- Palifermin, Lupron, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 82 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Chronic Gout
A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy II
This is one of two replicate randomized, double-blind, placebo-controlled, parallel arm trials to determine the safety and efficacy of two different dose levels of SEL-212 compared to placebo.
- Trial name
- DISSOLVE II (NCT04596540)
- Testing
- SEL-212 low-dose, SEL-212 high-dose
- Where
- 53 sites in United States, Georgia and 3 more
- Volunteers
- 153 people
- Sponsor
- Swedish Orphan Biovitrum
Macrophage Activation Syndrome (MAS), Still Disease, Juvenile Onset
Assessment of Macrophage Activation syndromE in STill's Disease in Italy
Assessment of macrophage activation syndrome in Still's disease: retrospective chart analysis of patient history, symptom resolution and treatment characteristics in Italy
- Conditions
- Macrophage Activation Syndrome (MAS), Still Disease, Juvenile Onset, Still's Disease, Adult-Onset
- Trial name
- AMETISTA (NCT06992505)
- Where
- 2 sites in Italy
- Volunteers
- 32 people
- Sponsor
- Swedish Orphan Biovitrum
Transplant-Associated Thrombotic Microangiopathy
A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.
- Trial name
- NCT05148299
- Testing
- Pegcetacoplan
- Where
- 14 sites in United States, France and 3 more
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Cold Agglutinin Disease
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
The purpose of the study is to determine the efficacy of pegcetacoplan administration compared to placebo in increasing hemoglobin (Hgb) level from baseline and avoiding transfusion in participants with primary cold agglutinin…
- Trial name
- NCT05096403
- Testing
- Pegcetacoplan
- Where
- 44 sites in United States, Austria and 12 more
- Volunteers
- 24 people
- Sponsor
- Swedish Orphan Biovitrum
B-Cell Non-Hodgkin Lymphoma
Anakinra for the Prevention of Cytokine Release Syndrome and Neurotoxicity in Patients With B-Cell Non-Hodgkin Lymphoma Receiving CD19-Targeted CAR-T Cell Therapy
This phase II trial studies how well anakinra works in decreasing the occurrence of cytokine release syndrome (CRS) and damage to the nerves (neurotoxicity) in patients with B-cell non-Hodgkin lymphoma who are receiving CD-19…
- Trial name
- NCT04359784
- Testing
- Anakinra, X-Ray Imaging, Positron Emission Tomography
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Fred Hutchinson Cancer Center
Hemophilia B
An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
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-MORE (NCT03901755)
- Testing
- Alprolix
- Where
- 29 sites in Czechia, Greece and 7 more
- Volunteers
- 151 people
- Sponsor
- Swedish Orphan Biovitrum
Chronic Gout
A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy
This is one of two replicate randomized, double-blind, placebo-controlled, parallel arm trials to determine the safety and efficacy of two different dose levels of SEL-212 compared to placebo.
- Trial name
- DISSOLVE I (NCT04513366)
- Testing
- SEL-212 low-dose, SEL-212 high-dose, Normal Saline
- Where
- 40 sites in United States
- Volunteers
- 112 people
- Sponsor
- Swedish Orphan Biovitrum
Immune Thrombocytopenia
ITP Registry and Accompanying Biospecimen Collection
The objective of this ITP registry is to collect clinical information, including biosampling, from consenting patients with a variety of ITPs at different points in the course of their disease.
- Trial name
- ITP-Registry (NCT05152238)
- Where
- 64 sites in Germany
- Volunteers
- 1,100 people
- Sponsor
- Jena University Hospital
Immune Thrombocytopenia, ITP
An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP
This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge…
- Trial name
- ADOPT (NCT04943042)
- Testing
- Avatrombopag
- Where
- 55 sites in Croatia, Czechia and 7 more
- Volunteers
- 199 people
- Sponsor
- Swedish Orphan Biovitrum
Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset
A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD)
A study to demonstrate efficacy and safety of anakinra in pediatric and adult Japanese patients with Still's disease (Systemic juvenile idiopathic arthritis [SJIA] and Adult-onset Still's disease [AOSD]).
- Trial name
- NCT05814159
- Testing
- Anakinra
- Where
- 8 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum