Clinical trials: Bleeding Diathesis
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.
Chronic Myeloproliferative Disorders, Leukemia
Monoclonal Antibody Therapy in Treating Patients With Advanced Cancer
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00003082
- Testing
- monoclonal antibody A27.15, monoclonal antibody E2.3
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Arizona
Haemophilia A and B
TFPI Levels in Haemophilia A and B Patients
The goal of this study is to measure the TFPI plasma level, a molecule involved in the regulation of the coagulation system, in haemophilia patients.
- Trial name
- NCT04570696
- Testing
- NON INTERVENTIONAL
- Where
- France
- Volunteers
- 100 people
- Sponsor
- CHU de Reims
Immune Thrombocytopenia
Immunomodulation With Romiplostim in Young Adults With ITP
The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it.
- Trial name
- iROM (NCT02760251)
- Testing
- romiplostim
- Where
- 5 sites in Switzerland
- Volunteers
- 15 people
- Sponsor
- University Hospital, Basel, Switzerland
Telemedicine, Exercise
Feasibility of a Blended Therapy Approach
Exercise is an important part of therapy guidelines in the rehabilitation of rare diseases (RDs) as Haemophilia and Myositis. The aim of this study is not to evaluate a new therapy intervention, but to evaluate the delivery of…
- Conditions
- Telemedicine, Exercise, Feasibility, Myositis
- Trial name
- bletheRD (NCT03713151)
- Testing
- Dividat FIT
- Where
- Switzerland
- Volunteers
- 30 people
- Sponsor
- University of Zurich
Genetic Linkage, Myelofibrosis
Genetic Analysis of Gray Platelet Syndrome
This study will identify and characterize the gene or genes responsible for Gray Platelet syndrome (GPS). Platelets are small blood cells that stick on injured blood vessels to form a plug and stop bleeding.
- Trial name
- NCT00069680
- Where
- United States
- Volunteers
- 123 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Hemophilia, Chronic Pain
Multimodal Telerehabilitation Versus Face-to-Face Care for Adults With Hemophilia and Chronic Pain
Hemophilia is an inherited bleeding disorder that is commonly associated with chronic joint pain, reduced physical function, and decreased quality of life.
- Trial name
- HEMO-TELE (NCT07682519)
- Testing
- other, Exercise
- Where
- Chile
- Volunteers
- 24 people
- Sponsor
- University of Chile
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
Haemophilia A
A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A.
The main objectives of this study are: to assess Optivate® consumption (IU/kg consumed per month for prophylactic and on-demand therapy and dose at each bleed). to assess clinical outcome when treating a bleed with Optivate®.
- Trial name
- NCT02246894
- Testing
- Optivate® (Human Coagulation Factor VIII)
- Where
- 5 sites in Poland
- Sponsor
- Bio Products Laboratory
Liver Cirrhosis
A Phase 2 Study of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia
This is a phase 2 study to evaluate the efficacy and safety of foscenvivint in patients with liver cirrhosis resulting from HIV/HCV co-infection in the setting of hemophilia.
- Trial name
- OP-724-H202 (NCT07681089)
- Testing
- Foscenvivint
- Where
- 4 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Kiminori Kimura, MD
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 Arthropathy
Physiotherapy and Reaction Time in Hemophilia
This randomised controlled study was conducted to investigate the comprehensive physiotherapy combined with home exercises on individuals with hemophilic arthropathy in the lower extremity.
- Trial name
- NCT06331091
- Testing
- Home exercise, Manual therapy, Neuromuscular electrical stimulation
- Where
- Turkey (Türkiye)
- Volunteers
- 21 people
- Sponsor
- Yuzuncu Yil University
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
Haemophilia B
A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Undergoing Major Surgery.
The main objective of the study is to investigate the safety and efficacy of Replenine®-VF administered by continuous infusion in appropriate dosage to prevent bleeding and achieve haemostasis in patients with haemophilia B…
- Trial name
- NCT02250560
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 5 sites in United Kingdom
- Sponsor
- Bio Products Laboratory
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