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Hemophilia A clinical trials

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NCT ID: NCT05044845 Recruiting - Hemophilia B Clinical Trials

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Start date: January 18, 2022
Phase:
Study type: Observational

Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low. Transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. Current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. In addition, few educational resources to explain basic gene therapy concepts exist in languages other than English. This study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries. Primary Objective: To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally Secondary Objectives: 1. To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally 2. To explore healthcare workers beliefs and attitudes about gene therapy 3. To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.

NCT ID: NCT05036278 Recruiting - Hemophilia A Clinical Trials

Prophylaxis Regimen for Hemophilia A Patients

PREDICT
Start date: July 28, 2022
Phase: Phase 4
Study type: Interventional

Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly. People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have become available. These products last longer in the body so that they require to be given less often with injections every 3-5 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on their experience. The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant. All participants will receive Jivi for 6 months. In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans: - participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose - participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose - participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome. This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

NCT ID: NCT05022459 Recruiting - Hemophilia A Clinical Trials

Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

STEP
Start date: August 16, 2023
Phase:
Study type: Observational

Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process. This disorder occurs mostly in males and in severe cases causes frequent bleeding episodes in joints and muscles which can lead to progressive damage that affects mobility and quality of life. Prophylactic FVIII administered intravenously every other day has been the standard of care treatment for HA for the past few decades. Sports and physical activity are generally encouraged in patients with hemophilia on appropriate prophylactic treatment to increase strength, prevent or decrease obesity, accrue and maintain bone density and encourage normal socialization. To ensure safety with participation in sports in persons with hemophilia A (PWHA), timing of FVIII administration is often adjusted to maximize FVIII at the time of sports. The exact factor level that is needed to safely participate in sports and minimize bleeding risk is not yet known. Based on clinical practice, infusion of FVIII to near the lower limit of normal right before participation in sports generally works to prevent bleeding. The study is looking at how well the newly approved medication Emicizumab works compared to Factor VIII to prevent bleeding in patients with Hemophilia A who play sports. The study will enroll children and adolescents who are already on Emicizumab or Factor VIII who are currently playing sports.

NCT ID: NCT04845555 Recruiting - Hemophilia A Clinical Trials

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

AIM-Active
Start date: September 1, 2019
Phase:
Study type: Observational

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

NCT ID: NCT04817462 Recruiting - Clinical trials for Hemophilia A, Severe

Liver Biopsy In Haemophilia Gene Therapy

Start date: August 5, 2022
Phase:
Study type: Observational

To perform a liver biopsy in haemophilia A and B patients stably expressing human FVIII/FIX for a period of at least 6 months following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.

NCT ID: NCT04805801 Recruiting - Clinical trials for Hemophilia A With Inhibitor

The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors

Start date: August 28, 2019
Phase:
Study type: Observational [Patient Registry]

To evaluate Safety and efficacy and pharmacokinetics, FVIII Inhibitor titers of Hemlibra subcutaneous injection (SC inj.) in Korean Hemophilia A patients with/without FVIII Inhibitors.

NCT ID: NCT04805021 Recruiting - Hemophilia A Clinical Trials

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

IMMUNHEMAC
Start date: November 30, 2021
Phase:
Study type: Observational

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.

NCT ID: NCT04775888 Recruiting - Clinical trials for Health Services Accessibility

Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies

PHAREO
Start date: September 21, 2020
Phase:
Study type: Observational

The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. To date, medications are exclusively dispensed in hospital pharmacies to ensure traceability and safety. This retrocession imposes accessibility constraints on patients and on their caregivers, increasing the burden of the disease, particularly in the organization of personal and professional daily life. The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in the Auvergne-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

NCT ID: NCT04768699 Recruiting - Clinical trials for Hemophilia A With Inhibitor

Study of Recombinant Human Coagulation Factor VIIa for Injection (FⅦa) in Patients With Hemophilia.

Start date: December 1, 2020
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT04747964 Recruiting - Hemophilia Clinical Trials

A Safety Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Start date: January 15, 2020
Phase: Phase 1
Study type: Interventional

This study will assess the pharmacokinetics and pharmacodynamics of STSP-0601 at five dose levels. The results will help identify the most optimal doses to treat bleedings in hemophilia patients with inhibitors.