Active clinical trials for "Hemophilia A"

The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice

A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.

Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and World Federation of Hemophilia (WFH). The efficacy and safety of prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been confirmed in well-designed clinical studies.To keep the factor level above 1%, the standard dosage for patients with severe hemophilia A is 20-40 Units /kg/infusion (average 30 Units /kg) every other day or three times a week. This dosage has a very high consumption of factor, up to 5000-6000 international unit(IU)/kg/year. The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world. But after the Recombinant Human Coagulation Factor VIII for injection (ADVATE) Patient Assistance Program(Golden Key) was launched in 24 Apr 2014 in Nanjing China, the affordability of patients was solved and many patients will get more chance to receive standard prophylaxis. This study is designed to evaluate the Annual Bleeding rate (ABR), joint health outcomes and QoL outcomes in subjects using standard prophylaxis under the conditions of routine practice.

Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate. Physicians complete a retrospective chart review on each enrolled patient. Patients will complete a one-time study questionnaire.

Participants will be assigned to A or B groups with a scale of 1:1 based on a prospectively randomized treatment-sequence assignment, i.e. infuse SCT800 followed by Xyntha (group A), or the alternate sequence (group B). All participants who completed the SCT800HA1 study will enter the efficacy and safety study (Protocol No.: SCT800HA3).

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high responding inhibitors and poor prognosis for success.

This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or Jivi. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

This study investigates the effect of exercise training on pain, physical activity and quality of life in pediatric hemophilia patients.

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.