Clinical trials: Moderate Hemophilia A
Hemophilia A
Study of a Gene Therapy Treatment for Hemophilia A
The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.
- Trial name
- KEYSTONE 1 (NCT06297486)
- Testing
- SPK-8011
- Where
- 27 sites in United States
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Severe Hemophilia A With Inhibitor
Thrombin Generation Assay (TGA) as Predictive Test for Haemostatic. Effectiveness of FVIII Concentrates in Haemophiliac A With Inhibitors
This is an observational, prospective, longitudinal, multicenter, cohort study designed with the scope to verify whether or not TGA may predict effectiveness of different FVIII concentrates class (devoid or rich of VWF) in…
- Trial name
- PredicTGA (NCT01505946)
- Testing
- TGA (Thrombin generation Assay)
- Where
- 15 sites in Italy
- Volunteers
- 25 people
- Sponsor
- Grifols Italia S.p.A
Haemophilia A (Moderate or Severe)
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study…
- Trial name
- LIBERTY (NCT06716814)
- Testing
- Efanesoctocog alfa
- Where
- 20 sites in Bulgaria, France and 5 more
- Volunteers
- 89 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and World Federation of…
- Trial name
- NCT02282410
- Testing
- ADVATE
- Volunteers
- 15 people
- Sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Hemophilia
Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia
The primary goal of this observational study is to determine if: health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly…
- Trial name
- NCT06191068
- Where
- Canada
- Volunteers
- 190 people
- Sponsor
- The Hospital for Sick Children
Hemophilia B
BAX 326 Surgery Study in Hemophilia B Patients
The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency…
- Trial name
- NCT01507896
- Testing
- Recombinant factor IX
- Where
- 14 sites in Argentina, Bulgaria and 8 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Haemophilia A, Haemophilia B
Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy
The purpose of this study intend to evaluate the effectiveness of functional orthoses (including custom-made insoles and shoes) for preventing and controlling repetitive haemarthrosis in patients suffering of haemophilic ankle…
- Trial name
- NCT00638001
- Testing
- custom-molded insoles and custom-molded shoes
- Where
- Belgium
- Volunteers
- 70 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia A
Goal Attainment and Physical Activity in People With Hemophilia A
This is a Phase 4, multi-center, observational study conducted in patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa in the US and Japan.
- Trial name
- ACTIVIIITY (NCT06530030)
- Testing
- efanesoctocog alfa
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Sanofi
Hemophilia A
Quality of Life Study of Helixate NexGen
The aim of this study is to describe Health Related Quality of Life (HRQoL) in adolescents and adults with Hemophilia A treated prophylactically or on-demand with Helixate NexGen.
- Trial name
- NCT01053715
- Where
- 31 sites in Austria, Belgium and 5 more
- Volunteers
- 78 people
- Sponsor
- CSL Behring
Hemophilia
KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous doses of KN057 in subjects with hemophilia A or B, with or without inhibitors to Factor VIII…
- Trial name
- NCT05421429
- Testing
- KN057 doseⅠ, KN057 dose Ⅱ, KN057 dose Ⅲ
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
Hemophilia A
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process.
- Trial name
- STEP (NCT05022459)
- Testing
- Emicizumab, FVIII
- Where
- 10 sites in United States
- Volunteers
- 72 people
- Sponsor
- Wayne State University
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
High Dose of Activated Recombinant Human Factor VII for Treatment of Mild/Moderate Joint Bleeds in Haemophilia Patients With Inhibitors
This trial is conducted in Europe. The aim of this trial is to evaluate the efficacy and safety of two dose schedules of activated recombinant human factor VII in treatment of joint bleeds in haemophilia patients with inhibitors.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT00571584
- Testing
- activated recombinant human factor VII
- Where
- 18 sites in France, Hungary and 5 more
- Volunteers
- 24 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Von Willebrand Disease
Efficacy and Safety of IL-11 in DDAVP Unresponsive
The purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP.
- Trial name
- IL-11DDAVP (NCT00994929)
- Testing
- Neumega (Oprelvekin, Interleukin 11, IL-11)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Pittsburgh
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche