Clinical trials: Blood Protein Disorders
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
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
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
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
Multiple Myeloma
Effect of AT7519M Alone and AT7519M Plus Bortezomib in Patients With Previously Treated Multiple Myeloma
The purpose of this study is to determine whether AT7519M alone or AT7519M plus bortezomib are effective treatments in patients with previously treated multiple myeloma.
- Trial name
- NCT01183949
- Testing
- AT7519M, Bortezomib
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- Astex Pharmaceuticals, Inc.
Neoplasms, Plasma Cell, Neoplasms by Histologic Type
A Study to Evaluate the Safety, PK/PD of (OriCAR-017) in Subjects With RR/MM - RIGEL Study
The is a first clinical study for Oricell Therapeutics Inc. in the United States to evaluate the safety, PK, PD and preliminary efficacy of our anti-GPRC5D cell product (OriCAR-017) in subjects with relapsed/refractory multiple…
- Conditions
- Neoplasms, Plasma Cell, Neoplasms by Histologic Type, Neoplasms, Hemostatic Disorders
- Trial name
- NCT06271252
- Testing
- OriCAR-017
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- OriCell Therapeutics Co., Ltd.
Multiple Myeloma
A Study of KW-2478 in Combination With Bortezomib in Subjects With Relapsed and/or Refractory Multiple Myeloma
The purpose of this study is to assess the safety and benefits of the investigational study drug, KW-2478, when given with bortezomib (Velcade®), a drug approved for the treatment of Multiple Myeloma (MM).
- Trial name
- NCT01063907
- Testing
- KW-2478, Bortezomib
- Where
- 26 sites in United States, Philippines and 1 more
- Volunteers
- 95 people
- Sponsor
- Kyowa Kirin Co., Ltd.
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
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe…
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A
- Trial name
- NCT03876301
- Testing
- Standard of Care FVIII Replacement therapy
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Multiple Myeloma, Neoplasm, Plasma Cell
Natural Killer Cell (CYNK-001) Infusions in Adults With Multiple Myeloma
This study will find the maximum tolerated dose (MTD) of CYNK-001 which contain NK cells derived from human placental CD34+ cells and culture-expanded. CYNK-001 cells will be given post Autologous Stem Cell Transplant (ASCT).
- Conditions
- Multiple Myeloma, Neoplasm, Plasma Cell, Neoplasms by Histologic Type, Neoplasms
- Trial name
- NCT04309084
- Testing
- CYNK-001
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Celularity Incorporated
Cancer, Hematologic Malignancies
Denosumab Compared to Zoledronic Acid in the Treatment of Bone Disease in Patients With Multiple Myeloma
The purpose of this study is to determine if denosumab is non-inferior to zoledronic acid in the treatment of bone disease from multiple myeloma.
- Conditions
- Cancer, Hematologic Malignancies, Multiple Myeloma, Oncology
- Trial name
- NCT01345019
- Testing
- Denosumab, Zoledronic acid, Denosumab (for the open-label treatment phase)
- Where
- 309 sites in United States, Australia and 27 more
- Volunteers
- 1,718 people
- Sponsor
- Amgen
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular…
- Trial name
- ATLAS-INH (NCT03417102)
- Testing
- fitusiran, Bypassing agents
- Where
- 58 sites in United States, Australia and 15 more
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Acquired Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.
- Trial name
- NCT01178294
- Testing
- OBI-1
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
Multiple Myeloma
Study of Vorinostat (MK-0683) or Placebo, in Combination With Bortezomib in Participants With Multiple Myeloma (MK-0683-088 AMN)
Study of the efficacy and safety of bortezomib administered in combination with vorinostat in patients with relapsed or refractory multiple myeloma.
- Trial name
- NCT00773747
- Testing
- Vorinostat, bortezomib
- Volunteers
- 637 people
- Sponsor
- Merck Sharp & Dohme LLC
Multiple Myeloma, Neoplasms
Continuous Lenalidomide Therapy Versus Observation Following Induction Without Lenalidomide, Pomalidomide or Thalidomide in Myeloma
The purpose of this study is to see how long lenalidomide therapy can maintain or improve the disease response obtained after induction therapy that does not include lenalidomide, pomalidomide or thalidomide; and consequently…
- Conditions
- Multiple Myeloma, Neoplasms, Plasma Cells, Paraproteinemias
- Trial name
- NCT02155634
- Testing
- Lenalidomide
- Sponsor
- Celgene
Coagulation Protein Disorders, Blood Loss, Surgical
Observational Study of Prophylaxis and Treatment of Acute Perioperative Bleeding With Beriplex® P/N (Probe Study)
Beriplex® P/N is made from human plasma (the liquid part of the blood) and contains the coagulation factors II, VII, IX and X which are important for blood clotting (coagulation).
- Conditions
- Coagulation Protein Disorders, Blood Loss, Surgical, Perioperative Care
- Trial name
- NCT01053169
- Testing
- Beriplex® P/N, Fresh Frozen Plasma (FFP) and Beriplex® P/N, Fresh Frozen Plasma
- Where
- 6 sites in United Kingdom
- Volunteers
- 445 people
- Sponsor
- CSL Behring