Clinical trials: Hereditary Factor VIII Deficiency Disease With Inhibitor
Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor
Pharmacokinetic-guided Dosing of Emicizumab
The goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of…
- Conditions
- Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor, Hemophilia A, Severe, Adolescent
- Trial name
- DosEmi (NCT06320626)
- Testing
- Emicizumab - PK-guided dose reduction, Emicizumab - Dosis continuation group, Emicizumab - Dose adjustment group
- Where
- 8 sites in Netherlands
- Volunteers
- 95 people
- Sponsor
- Kathelijn Fischer
Hemophilia A Without Inhibitor
The Hemophilia Inhibitor Prevention Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
- Trial name
- NCT04303559
- Testing
- Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors
The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use.
- Trial name
- NCT06312475
- Testing
- KN057
- Where
- China
- Volunteers
- 53 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia A With Inhibitor
Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction
The goal of this clinical trial is to learn how well Altuviiio (efanesoctacog alpha) works for Immune Tolerance Induction (ITI) while using Hemlibra (emicizumab) to prevent bleeds.
- Trial name
- UTILITI (NCT07158606)
- Testing
- efanesoctacog alpha, Emicizumab
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Versiti Blood Health
Hemophilia A
Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug.
- Trial name
- AFEEL (NCT06938659)
- Testing
- Emicizumab Injection, Factor VIII (FVIII)
- Where
- Bangladesh
- Volunteers
- 20 people
- Sponsor
- Dhaka Medical College
Hemophilia A With Inhibitor
ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor
To evaluate the time of response, sustained remission rate, and relapse rate of CD38 monoclonal antibody (Daratumumab) combined with SCT800 (rFVIII) in the treatment of hemophilia A adolescents and adults with high titer…
- Trial name
- NCT05888870
- Testing
- SCT800 and Daratumumab, SCT800
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk…
- Trial name
- NCT04647227
- Testing
- coagulation factor VIIa [recombinant]-jncw
- Where
- 21 sites in United States
- Volunteers
- 55 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A With Inhibitor
Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled.
- Trial name
- HA-LD-ITI (NCT03598725)
- Testing
- Coagulation Factor VIII, Prednisone, Rituximab
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Children's Hospital
Hemophilia A With Inhibitor
An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
This study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema…
- Trial name
- EmiandMe (NCT04723693)
- Testing
- Qualitative Interview
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Haemnet
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B
Phase 2, multi-center, open-label study designed to evaluate the PK, bioavailability, PD, efficacy and safety of a daily subcutaneous [SC] treatment regimen with MarzAA for bleeding prophylaxis in 12 adult subjects with…
- Trial name
- NCT03407651
- Testing
- Coagulation Factor VIIa variant
- Where
- 9 sites in Armenia, Georgia and 3 more
- Volunteers
- 11 people
- Sponsor
- Catalyst Biosciences
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A With Inhibitor
The Hemophilia Inhibitor Eradication Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.
- Trial name
- NCT04303572
- Testing
- Eloctate ITI, Emicizumab
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Factor VII Deficiency, Glanzmann Thrombasthenia
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Conditions
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- Trial name
- NCT04548791
- Testing
- Coagulation Factor VIIa variant
- Where
- 20 sites in United States, India and 3 more
- Volunteers
- 19 people
- Sponsor
- Catalyst Biosciences
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII.
- Trial name
- GENEr8-INH (NCT04684940)
- Testing
- Valoctocogene roxaparvovec
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 10 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A With Inhibitor, Hemophilia A
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA).
- Trial name
- NCT06357572
- Testing
- Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Additional coagulation tests
- Where
- Poland
- Volunteers
- 6 people
- Sponsor
- Enzyre B.V.
Hemophilia A With Inhibitor
Hemophilia Inhibitor Genetics Study (HIGS)
Several non-genetic and genetic factors that could influence the risk of inhibitor development in hemophilia A have been discussed but not fully explored. The aim of the HIGS is to identify these genetic factors.
- Trial name
- NCT00166387
- Testing
- Blood draw
- Where
- Sweden
- Volunteers
- 1,137 people
- Sponsor
- Skane University Hospital
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Study of Recombinant Human Coagulation Factor VIIa for Injection (FⅦa) in Patients With Hemophilia.
Aim of this trial is to assess the pharmacokinetics and pharmacodynamics (PK/PD) of recombinant human coagulation factor VIIa for injection (FⅦa) in patients with hemophilia.
- Trial name
- NCT04768699
- Testing
- TQG203, TQG203/NovoSeven®
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Hemophilia a with Inhibitor
Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)
Hemophilia A is a blood coagulation disorder caused by deficient or dysfunctional clotting factor VIII (FVIII) leading to incomplete haemostasis.
- Trial name
- NCT06864975
- Testing
- Advate®
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Runhui WU
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
Hereditary Factor VIII Deficiency Disease With Inhibitor
A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors.
- Trial name
- BPATXAS (NCT01800435)
- Testing
- aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA
- Where
- Norway
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oslo University Hospital