Clinical trials: Coagulation Protein Disorders
Severe Hemophilia A
Canadian Hemophilia Prophylaxis Study
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis…
- Trial name
- CHPS (NCT01085344)
- Testing
- Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
- Where
- Canada
- Volunteers
- 56 people
- Sponsor
- The Hospital for Sick Children
Von Willebrand Diseases
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy…
- Trial name
- NCT04052698
- Testing
- Wilate
- Where
- 14 sites in United States, Belarus and 6 more
- Volunteers
- 43 people
- Sponsor
- Octapharma
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Hemophilia A
BAX 802 in CHA With Inhibitors
The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.
- Trial name
- CHAWI (NCT02895945)
- Testing
- Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)
- Where
- 24 sites in United States, Bulgaria and 10 more
- Volunteers
- 8 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Primary Objective: To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-Kids (NCT04759131)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 40 sites in United States, Australia and 13 more
- Volunteers
- 74 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
- Trial name
- NCT04396639
- Testing
- Moroctocog-alfa (AF-CC)
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Chronic Myeloproliferative Disorders, Leukemia
Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
RATIONALE: Giving chemotherapy, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- LCCC0510 (NCT00448357)
- Testing
- rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Hemophilia B
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Severe Hemophilia A
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Trial name
- NCT01125813
- Testing
- recombinant Factor VIII
- Where
- 11 sites in Austria, Bulgaria and 2 more
- Volunteers
- 32 people
- Sponsor
- Octapharma
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Hemophilia, Hemophilic Arthropathy
Platelet-Rich Plasma Intra-Articular Injection in Treating Hemophilic Arthropathy
Severe hemophilia is characterized by frequent and lifelong bleeding, with more than 60% of bleeds occurring into joints . Repeated joint bleeding leads to chronic synovitis, cartilage damage and bony destruction.
- Trial name
- NCT02601170
- Testing
- Platelet-Rich Plasma Intra-Articular Injection, Hyaluronic Acid Viscosupplementation
- Volunteers
- 22 people
- Sponsor
- Tri-Service General Hospital (TSGH)
Gingival Bleeding, Von Willebrand Diseases
Gingival Bleeding and Von Willebrand Disease Typ 2 and 3
Von Willebrand disease (VWD) is the most common inherent bleeding disorder resulting in prolonged bleeding time. Gingival bleeding is a frequently reported symptom of VWD.
- Trial name
- NCT03078595
- Where
- 2 sites in Germany
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Goethe University
Hemophilia B
BAX 326 Pediatric Study
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
- Trial name
- NCT01488994
- Testing
- BAX326
- Where
- 11 sites in India, Poland and 4 more
- Volunteers
- 23 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A.
- Trial name
- ASPIRE (NCT01454739)
- Testing
- rFVIIIFc
- Where
- 78 sites in United States, Australia and 20 more
- Volunteers
- 240 people
- Sponsor
- Bioverativ Therapeutics Inc.
Antithrombin III Deficiency
Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate
To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.
- Trial name
- NCT00319228
- Testing
- Plasma-derived AT-III concentrate
- Volunteers
- 3 people
- Sponsor
- Instituto Grifols, S.A.
Hemophilia A
A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with…
- Trial name
- HAVEN 2 (NCT02795767)
- Testing
- Emicizumab
- Where
- 28 sites in United States, Costa Rica and 8 more
- Volunteers
- 88 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
ADYNOVATE Drug Use-Results Survey
The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients: 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT03169972
- Testing
- ADYNOVATE
- Where
- 52 sites in Japan
- Volunteers
- 135 people
- Sponsor
- Takeda
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00544115
- Testing
- busulfan, cyclophosphamide, cyclosporine
- Volunteers
- 260 people
- Sponsor
- City of Hope Medical Center