View clinical trials related to Hemophilia A.
Filter by: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 (FVIII) or Factor IX (FIX). 24 adult participants 18 to 70 years of age with moderately severe to severe hemophilia A or hemophilia B (defined as FVIII or FIX activity ≤2%, respectively) with or without inhibitors (including 18 HA/HB patients without inhibitors and 6 HA/HB patients with inhibitors) are expected to be enrolled in this study during which they will receive prophylaxis treatment (defined as treatment by SC injection once weekly of KN057).
People with hemophilia A do not have enough of a protein found naturally in the blood called "clotting factor 8", also known as FVIII. This protein helps the blood to clump together to prevent and stop bleeding. People with lower levels of FVIII or FVIII that does not work properly may bleed for a long time from minor wounds, bleed into their joints, or have internal bleeding. The study treatment, Jivi (also called damoctocog alfa pegol), is already available as a treatment for people aged 12 years and older with hemophilia A, to help prevent bleeding, also known as "prophylactic" treatment. It works by replacing the missing FVIII, or the FVIII that does not work properly. It can also be used to stop bleeding that has already occurred and prior to surgery to prevent bleeding. The main goal of this study is to learn how damoctocog alfa pegol is used in the "real world" as a treatment in the United States (US) and how well it works and what other treatments patients use while receiving damoctocog alfa pegol treatment. It will also determine how satisfied people are with the treatment. There will be no required visits with a study doctor in this study. The study will include about 20 male or female patients in the US aged 12 years and over who have hemophilia A. All the patients in this study will have switched from their previous FVIII replacement treatment to damoctocog alfa pegol. While the patients are receiving damoctocog alfa pegol, they will complete a survey to say how they feel about the treatment. Their doctors will also record information about their treatment with damoctocog alfa pegol and how well it is working. This study will collect information from the patients' medical records and surveys. They will use this information to find out more about treatment with damoctocog alfa pegol under "real world" conditions. They will look at: - how often the patients receive damoctocog alfa pegol and how much they use - what other treatments the patients received before receiving damoctocog alfa pegol, how they used it and how much they used - how well damoctocog alfa pegol works at preventing bleeding, and how it compares to previous products used.
The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP. This is a medicine that doctors can already prescribe in other countries. The medicine will be injected into a vein (intravenous injection). At the visits to the clinic, the medicine will be injected by the study doctor. When treating themselves at home, participants inject the medicine using a needle and vial set. The study will last for about 12-16 months. The participants will have between 9 and 19 visits to the clinic and possibly also some phone calls with the study doctor. At all visits to the clinic, the participants will have blood samples taken.
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).
This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated limitations imposed on those with severe haemophilia may not translate to lessen the biomedical burden of living with a history of severe haemophilia. We wish to explore these issues further in the Learning to Live study. The study will also seek to identify the ongoing support needs of those who transition to a milder bleeding phenotype.
This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeds. Mim8 will be injected with a thin needle into the skin. The study will last for about 54-98 weeks, from screening to follow-up visit, In case the participant experiences bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor.
This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. This is a pilot preclinical study.
Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen. Methods A single-centre prospective cohort study, the investigators recruited consecutive eligible patients aged 5-25 years with clinically severe haemophilia A (FVIII:C ≤3%), no inhibitor, on low-dose weight-based prophylaxis at King Chulalongkorn Memorial Hospital (KCMH) from July 2021 to February 2022. All of patients with clinically severe haemophilia A received low dose weight-based standard half-life FVIII concentrates replacement prophylaxis for ≥ 1 year prior to enrolment in the study. The data of annual bleeding rate (ABR), annual joint bleeding rate (AJBR), annual FVIII use (prophylactic and breakthrough bleeding dosing) in the last 6 months before the study and number of target joints were collected at the beginning of the study. Baseline variables, including age and weight, were recorded before performing the analyses using online medical device (www.mypkfit.com). Wash-out period for 72 hours, each participant subsequently received a dose of 20 IU/kg FVIII by intravenous injection. Blood samples were collected and the concentration of FVIII was measured two times at 3 h and 48 h or 72 h after injection by one-stage technique. Desired FVIII trough levels were selected in this study as 1%. Individually proper regimen were selected by discussion with patients and families. All of participant individually underwent dose calculation of EHL factor VIII concentrates and received low dose PK-guided regimen (10-20u/kg, 2-3times/week) with EHL FVIII concentrates for 6 months. If breakthrough bleeding occurs, FVIII concentrates 500 U intravenous injection immediately. ABR, AJBR, HJHS and annual FVIII concentrates use were again prospectively recorded during intervention period after PK adjustment for 6 months. Primary objectives To compare clinical outcomes including annual bleeding rate (ABR), annual joint bleeding rate (AJBR) and Haemophilia joint health score (HJHS) before and after switching from standard half-life (SHL) to Extended half-life (EHL) factor VIII concentrates with adjusted dosing by PK-guided program (MyPKFiT®) in severe haemophilia A patients Secondary objectives To compare factor VIII concentrates consumption before and after using PK-guided program (MyPKFiT®) adjusting dose of factor VIII infusion in severe HA patients.
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast