Clinical trials: Hemophilia A With Inhibitors
Hemophilia a, Hemophilia B
The Safety and Tolerability of SerpinPC in Healthy Men and in Men with Severe Blood Disorders (haemophilia a and B)
The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and…
- Trial name
- NCT04073498
- Testing
- SerpinPC
- Where
- 3 sites in Georgia, Moldova and 1 more
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- ApcinteX Ltd
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Non-Interventional Study of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors
This study is conducted in Japan. The aim of this registry study is to observe the use of single dose and multi-dose use of eptacog alpha (NovoSeven®) and to compare short-term outcomes, including effectiveness, safety, quality…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01586936
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 9 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia A With Inhibitors
A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5)
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A.
- Trial name
- FRONTIER 5 (NCT05878938)
- Testing
- NNC0365-3769 (Mim8) PPX
- Where
- 36 sites in United States, Austria and 10 more
- Volunteers
- 61 people
- Sponsor
- Novo Nordisk A/S
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
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
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
Congenital Bleeding Disorder, Haemophilia A
Survey Evaluating the Psychosocial Effects of Living With Haemophilia
This study is conducted in Africa, Asia, Europe, North America and South America. The purpose of the survey is to identify the key psychosocial issues affecting patients with haemophilia.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Haemophilia A With Inhibitors
- Trial name
- HERO (NCT01322620)
- Testing
- No treatment given
- Where
- 3 sites in Canada, Spain and 1 more
- Volunteers
- 1,236 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia A and B
A Phase III Clinical Trial Evaluating SR604 Injection for Prophylactic Treatment in Patients With Hemophilia
This is a single-arm, multicenter Phase III clinical trial designed to evaluate the efficacy and safety of prophylactic treatment with SR604 Injection in hemophilia A/B patients with or without inhibitors.
- Conditions
- Hemophilia A, Hemophilia A and B, Hemophilia B
- Trial name
- NCT07876674
- Testing
- SR604
- Volunteers
- 90 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Hemophilia A
Early Prophylaxis Immunologic Challenge (EPIC) Study
The purpose of the study was to assess if a once-weekly prophylactic regimen of 25 IU/kg ADVATE started at or before 1 year of age and before the onset of a severe bleeding phenotype (ie, joint bleeding), together with the…
- Trial name
- EPIC (NCT01376700)
- Testing
- Recombinant antihemophilic factor, plasma/albumin-free method (rAHF-PFM)
- Where
- 19 sites in United States, Austria and 10 more
- Volunteers
- 22 people
- Sponsor
- Baxalta now part of Shire
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.
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Non-Interventional Study of NovoSeven® Used in Patients With Haemophilia A and B With Inhibitors Under Normal Clinical Practice
This study is conducted in Japan. The aim of this non-interventional study is to investigate the safety and effectiveness of treatment with eptacog alpha (NovoSeven®) under normal clinical practice conditions.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01579955
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 321 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
Patient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany
This study aims to generate structured patient preference evidence on prophylactic treatment options for haemophilia A for adults with and without inhibitors to support health technology assessment and benefit evaluation…
- Trial name
- NCT07653139
- Testing
- No treatment given
- Where
- Germany
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
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.
Congenital Bleeding Disorder, Haemophilia A
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
This study is conducted in the United States of America (USA). The aim of the study is to assess the impact of pain on functional impairment and quality of life in adult persons with hemophilia (PWH) with and without inhibitors…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT01988532
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 381 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A Without Inhibitor, Hemophilia B Without Inhibitor
Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors
The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B without inhibitors and is safe to use.
- Trial name
- NCT06569108
- Testing
- KN057
- Where
- China
- Volunteers
- 125 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
Study Evaluating Inhibitor Specificity in Hemophilia A
This investigation will utilize a systematic determination of anti-FVIII antibody specificity in PTPs (> 50 ED) with hemophilia A who have developed inhibitors in response to treatment with any FVIII product(s).
- Trial name
- NCT00151385
- Testing
- single blood draw
- Where
- 23 sites in United States, Belgium and 5 more
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use.
- Trial name
- explorer7 (NCT04083781)
- Testing
- Concizumab
- Where
- 99 sites in United States, Algeria and 27 more
- Volunteers
- 134 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors.
- Trial name
- NCT06703606
- Testing
- MARSTACIMAB
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Hemophilia A
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A…
- Trial name
- NCT04541628
- Testing
- SIG-001
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eli Lilly and Company