Clinical trials: Bleeding Diathesis
Congenital Bleeding Disorder, Haemophilia A
Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients
This trial is conducted in Europe and Japan. The aim of this trial is to assess the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of single doses of NNC128-0000-2011, when administered i.v.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01288391
- Testing
- NNC 0128-0000-2011
- Where
- 3 sites in Germany, Japan and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A
Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development
This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies.
- Trial name
- NCT01445197
- Testing
- Biostate
- Where
- 7 sites in Austria, Germany and 3 more
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Haemophilia A
Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared…
- Trial name
- NCT00245297
- Testing
- Kogenate FS, reconstituted in a suspension of liposomes
- Where
- 3 sites in Russia
- Volunteers
- 16 people
- Sponsor
- Recoly N.V.
Hemophilia A
Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
This Phase IV, multicenter study will evaluate whether participants with Hemophilia A (PwHA) with or without inhibitors receiving emicizumab prophylaxis can safely undergo minor surgical procedures without additional prophylactic…
- Trial name
- NCT03361137
- Testing
- Emicizumab
- Where
- 8 sites in United States
- Volunteers
- 14 people
- Sponsor
- Genentech, Inc.
Protein C Deficiency
Ceprotin Treatment Registry
The overall objective is to collect and assess data on the treatment, safety, and treatment outcomes of subjects prescribed, receiving and participating in the Ceprotin treatment registry.
- Trial name
- NCT01127529
- Testing
- Protein C Concentrate (Human)
- Where
- 28 sites in United States, Austria and 4 more
- Volunteers
- 43 people
- Sponsor
- Baxalta now part of Shire
Factor VIII Deficiency
A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Primary Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-1 (NCT04161495)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 47 sites in United States, Argentina and 17 more
- Volunteers
- 159 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe…
- Trial name
- NCT00623727
- Testing
- rFVIII-FS/pegylated liposomes (BAY79-4980), rFVIII-FS/WFI (BAY14-2222)
- Where
- 95 sites in United States, Argentina and 24 more
- Volunteers
- 143 people
- Sponsor
- Bayer
Chronic Thrombocytopenia, Immune Thrombocytopenia
Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)
Core Study: To demonstrate that the efficacy of avatrombopag (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult participants with chronic immune thrombocytopenia…
- Trial name
- NCT01438840
- Testing
- Avatrombopag, Standard of care
- Where
- 25 sites in Australia, Belgium and 9 more
- Volunteers
- 49 people
- Sponsor
- Eisai Inc.
Blood Coagulation Disorders, Hemophilia A
Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A
The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods).
- Trial name
- Leopold I (NCT01029340)
- Testing
- Recombinant Factor VIII (BAY81-8973), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 60 sites in United States, Argentina and 20 more
- Volunteers
- 74 people
- Sponsor
- Bayer
Hemophilia A
Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A
The purpose of this post-marketing safety study is to evaluate the safety, immunogenicity, and effectiveness of ADVATE in previously untreated patients (PUPs) in China with moderate to severe hemophilia A.
- Trial name
- NCT02634723
- Testing
- Octocog alfa (recombinant human coagulation factor VIII)
- Where
- 6 sites in China
- Volunteers
- 21 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Disease
Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
- Trial name
- NCT00941616
- Testing
- Biostate®
- Where
- 5 sites in Bulgaria, Poland and 2 more
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Hemophilia A
A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and…
- Trial name
- NCT05251090
- Testing
- ADVATE, FRSW107
- Where
- 6 sites in China
- Volunteers
- 13 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Primary Immune Thrombocytopenia
Efficacy and Safety of QL0911 in Adult Patients With Chronic Primary Immune Thrombocytopenia
QL0911, a recombinant human thrombopoietin mimetic peptide-Fc fusion protein for injection, is a romiplostim (Nplate®) biosimilar for the treatment of primary immune thrombocytopenia (ITP).
- Trial name
- NCT05621330
- Testing
- QL0911
- Where
- China
- Volunteers
- 216 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Hemophilia A
Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII
The ability of a new recombinant porcine coagulation factor VIII, B-domain deleted (called "OBI-1"), to control the non-life- or limb-threatening bleeding episodes patients with hemophilia A commonly develop is being evaluated.
- Trial name
- NCT00245245
- Testing
- recombinant porcine coagulation factor VIII (OBI-1)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Octagen Corporation
Immune Thrombocytopenia
Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)
Study in patients with persistent and chronic Immune Thrombocytopenia (ITP), who have failed to respond or relapsed after prior therapy, with a platelet count <30,000/µL.
- Trial name
- NCT04056195
- Testing
- SKI-O-703
- Where
- 27 sites in United States, Greece and 3 more
- Volunteers
- 61 people
- Sponsor
- Oscotec Inc.
Thrombocythemia, Essential, Primary Myelofibrosis
An Extension Study of Bomedemstat (IMG-7289/MK-3543) in Participants With Myeloproliferative Neoplasms (IMG-7289-CTP-202/MK-3543-005)
This is a multi-center, open-label extension study to assess the long-term safety and efficacy of bomedemstat administered orally once daily in participants with a Myeloproliferative Neoplasm (MPN) who participated in a prior…
- Trial name
- NCT05223920
- Testing
- Bomedemstat
- Where
- 19 sites in United States, Australia and 5 more
- Volunteers
- 81 people
- Sponsor
- Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
Hemophilia A
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
- Trial name
- NCT05936580
- Testing
- Nuwiq
- Where
- 16 sites in United States, Finland and 8 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Haemophilia B
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B.
- Trial name
- NCT03745924
- Testing
- Nonacog beta pegol
- Where
- 30 sites in Austria, Belgium and 11 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of…
- Trial name
- NCT01271868
- Testing
- IB1001
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 9 people
- Sponsor
- Medexus Pharma, Inc.