Clinical trials: Hereditary Factor VIII Deficiency Disease With Inhibitor
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
Male, Hemophilia A With Inhibitor
SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment
This is an open-label, multicenter Phase 1Ib/2II clinical trial of SS109 in adult hemophilia patients (≥ 18 years) with FVIII or FIX inhibitors to evaluate the PK/PD profile of SS109 and NovoSeven® after a single dose in adult…
- Conditions
- Male, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Hemophilia
- Trial name
- NCT06010953
- Testing
- SS109
- Where
- 9 sites in China
- Volunteers
- 24 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
A Phase I of SS109 in Hemophilia A or and B With Inhibitors
This phase I study aims to evaluate the safety, PK/PD and immunogenicity of SS109 in hemophilia A or and B with inhibitors. Twenty -seven patients are enrolled in study, and divided into five dose cohorts, from 30μg/kg to…
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency
- Trial name
- NCT05651061
- Testing
- SS109
- Where
- 10 sites in China
- Volunteers
- 27 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Severe Hemophilia A, Severe Hemophilia A With Inhibitor
Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy.
- Conditions
- Severe Hemophilia A, Severe Hemophilia A With Inhibitor, Severe Hemophilia A Without Inhibitor
- Trial name
- NAVIGATE (NCT05802836)
- Testing
- no interventions
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Christoph Königs
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
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
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, Hemophilia B With Inhibitor
Study on the Dose-response Relationship of Pharmacodynamic Parameters in Patients With Hemophilia With Inhibitors
Randomized, double-blind, single-dose, 5 ways crossover, exploratory clinical trial evaluating four different doses of AryoSeven (eptacog alfa, activated) and NovoSeven on selected pharmacodynamic parameters in patients with…
- Trial name
- NCT04789954
- Testing
- Eptacog alfa, activated
- Where
- Iran
- Volunteers
- 14 people
- Sponsor
- AryoGen Pharmed Co.
Hemophilia A With Inhibitor
A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab
Hemophilia A is a severe, life-long, genetic bleeding disorder characterized by a deficiency of factor VIII (FVIII), a crucial cofactor of the coagulation system.
- Trial name
- NCT04205175
- Testing
- Feiba
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
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
Severe Hemophilia A
Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform
This study will evaluate if Eloctate is superior to Emicizumab in reducing inhibitors in children with severe hemophilia when given before the first bleed (preemptive) and continued weekly to prevent bleeds (prophylaxis); and…
- Trial name
- INHIBIT (NCT02196207)
- Testing
- Eloctate Prophylaxis, Emicizumab Prophylaxis, Eloctate ITI plus Emicizumab
- Where
- United States
- Sponsor
- Margaret Ragni
Hemophilia A With Inhibitor
A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip
The purpose of this study is to demonstrate that PEGylated liposomes (PEGLip) can shield FVIII from the immune system and inhibitors, and therefore provide a prophylactic FVIII replacement therapy for patients with inhibitors to…
- Trial name
- NCT04592692
- Testing
- PEGylated Liposome (PEGLip)
- Where
- 5 sites in Russia
- Volunteers
- 20 people
- Sponsor
- Ascension Healthcare Development Limited
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
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, Hemophilia B
The Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia
- Conditions
- Hemophilia a, Hemophilia B, Hemophilia a with Inhibitor, Hemophilia B with Inhibitor
- Trial name
- PRESent-6 (NCT06568302)
- Testing
- SerpinPC
- Where
- 2 sites in Georgia, Moldova
- Volunteers
- 15 people
- Sponsor
- ApcinteX Ltd
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
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, Hemophilia B
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe…
- Conditions
- Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
- Trial name
- NCT04072237
- Testing
- MarzAA (marzeptacog alfa [activated])
- Where
- 5 sites in Bulgaria, Russia
- Volunteers
- 11 people
- Sponsor
- Catalyst Biosciences
Hemophilia A With Inhibitor, Hemophilia A
INdividualized ITI Based on Fviii(ATE) Protection by VWF
The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex…
- Trial name
- INITIATE (NCT03204539)
- Testing
- Wilate
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- University of California, Davis