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73 active trials for Hemophilia A

A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.

Start: March 2020

Emicizumab PUPs and Nuwiq ITI Study

This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI).

Start: May 2021

Health Related Quality of Life of Youth and Young Adults With Haemophilia A

Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.

Start: April 2021

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

Primary Objective: - To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment. To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. To evaluate the safety and tolerability of BIVV001 treatment. To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). To evaluate the efficacy of BIVV001 for perioperative management

Start: February 2021

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.

Start: April 2021

UPLC-MS/MS Monitoring of Emicizumab Therapy

Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.

Start: August 2021

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 4 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and Parts 1b, 2 and 3 will be conducted in haemophilia A & B patients in Moldova and Georgia. Part 1a of the study will look at how safe the drug is when given as single doses to healthy volunteers at different strengths and via 2 different routes of administration (through a vein or via an injection under the skin). Parts 1b, 2 and 3 of the study will look at the safety of the drug when given as an injection under the skin to patients with severe haemophilia A or B. The study will also investigate how the levels of the drug in the blood change over a period of time and how the drug acts in the body by taking blood samples. These blood samples will measure the concentration of the drug in the blood and measure certain aspects of the blood to determine how the drug affects them. The study sponsor (ApcinteX) is developing this drug for the treatment of haemophilia A and haemophilia B, which are 2 types of rare blood disorders which affect the body's ability to form blood clots. Patients who have haemophilia A and B do not have certain clotting factors in their blood which means that they experience difficulty in stopping bleeding after injury and can be prone to extended periods of bleeding. Current treatments for haemophilia involves injections which replace the missing factors in the blood. However these treatments are short term and therefore patients require regular treatments in order to manage the condition. Therefore, there is a need to develop more effective treatments which provide longer term benefits. The aim of SerpinPC is to prevent bleeding rather than to have to treat bleeds to minimise pain and damage after they have occurred.

Start: August 2019

A Study of BAX 888 in Male Adults With Severe Hemophilia A

The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults. Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their study clinic multiple times.

Start: March 2018

Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C?2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C?1%)

To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish a minimum of 6 months of prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to SB-525 capsid (AAV6), prior to the Phase 3 gene therapy study. The enrollment for hemophilia B participants is completed. At this time participants are only being enrolled for hemophilia A cohort.

Start: July 2018

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

The purpose of this study is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Start: February 2018

Acquired Hemophilia A and Autoimmunity. Study of Lymphocyte Populations and Myeloid-Derived Suppressor Cells

Acquired hemophilia A is a rare condition of hemostasis secondary to the development of antibodies against factor VIII. This is a potentially serious pathology that can be life-threatening due to the major risk of bleeding caused by the sometimes drastic decrease in the level of circulating factor VIII. This pathology occurs overwhelmingly in elderly subjects or, more rarely, in young women, during the postpartum period. It appears idiopathic in 50% of cases and associated, for the other cases, with underlying pathologies such as autoimmune pathologies (rheumatoid arthritis and bullous pemphigoid in particular) and neoplasias, or with a particular circumstance represented by the post -partum. The association between this autoimmune pathology and its association with pathologies of the same type or with circumstances involving the immune system, suggests that common mechanisms could favor its emergence. This study therefore proposes to study lymphocyte populations and subpopulations as well as Myeloid-Derived Suppressor Cells and the cytokine profile, which are abnormal in a large part of autoimmune pathologies.

Start: May 2021

Association Between Individual Clotting Factor Level Monitoring and the Risk of Bleeding Whilst Physical Active Conditions

The aim of this prospective multicentre study is to evaluate the influence of individual clotting factor level while being physically active

Start: September 2019

Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A

The primary objectives of the study are to further evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in adolescent and adult patients with hemophilia A.

Start: April 2021

An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A.

The primary objectives of the study are to evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the Prevention and Treatment of Bleeding in patients with hemophilia A. The secondary objectives are to evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the prevention and treatment of bleeding episodes, to investigate the quality of life in patients who used the FRSW107.

Start: October 2020

Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®

The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®

Start: November 2016

National Longitudinal Cohort of Hematological Diseases

Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China. Method The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, or hemophilia in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, as well as medical expense information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

Start: December 2020

Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability

Using anonymized patient data collected as part of the WAPPS-Hemo project to explore the sources of variability in individual pharmacokinetics (PK); use the sources of variability to improve the performance of the WAPPS-Hemo models through the addition of the predictors of PK variability as covariates.

Start: May 2018

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

Start: May 2021

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 the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes To assess changes in health-related quality of life (QOL) over time To characterize antithrombin (AT) reduction and thrombin generation (TG) increase To characterize the pharmacokinetics (PK) of fitusiran

Start: September 2015

Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A

The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough >1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.

Start: April 2019