Active clinical trials for "Hemophilia A"

Introduction: The common clinical manifestations of hemophilia are skeletal muscle bleeds, especially hematomas and hemarthrosis. Repeated episodes of joint bleeding in certain joints, causing a progressive joint deterioration. Secondary disorders to this joint degeneration include: biomechanical alterations, loss of range of movement and periarticular muscle atrophy. Design. A prospective, multicenter and longitudinal pilot study to evaluate the efficacy of a treatment protocol with fascial therapy applied in patients with hemophilic arthropathy of the elbow. Aimed: To evaluate the safety and efficacy of a physiotherapy treatment by fascial therapy in patients with hemophilic arthropathy of the elbow Patients: A total of 60 patients with hemophilia and prophylactic treatment will be recruited for inclusion in the study. Patients will be recruited in 6 centers, from different regions of Spain. Intervention: Each session will last approximately 50 minutes, with three physiotherapy sessions taking place over a period of 3 weeks. The treatment program includes 11 maneuvers that must be administered bilaterally: Measuring instruments and study variables: goniometric evaluation (range of movement); visual analog scale (joint pain); Haemophilia Joint Health Score (joint status); DASH Questionnaire (functionality of upper limbs); SF-36 Questionnaire (perception of quality of life). At the same time, the study will allow to determine joint bleeding caused by applied physiotherapy treatment. Expected results: First, it is intended to demonstrate the safety of this physiotherapy technique in patients with hemophilia. Likewise, an improvement in the perception of elbow pain and joint mobility is expected. An improved functionality of the upper limb is also foreseen and with it, an enhanced perception of quality of life of these patients.

This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients with haemophilia B.

One of the major shortcomings in studying bone disease in hemophilia is the lack of fracture outcome data demonstrating the clinical significance of decreased BMD and altered bone biomarkers in the hemophilia population. This study demonstrates that PwH have an increased risk of fracture compared to the general population and that the issue of bone health will increase in importance as the PwH population ages.

This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study. A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.

The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX

This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A. A minimum of 25 previously treated subjects ≥ 6 to < 12 years of age and at least 25 subjects < 6 years of age who have undergone > 50 exposure days (EDs) with a previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned to either an on-demand or prophylaxis treatment regimen and will receive rVIII-SingleChain at a dose to be determined by the investigator. Hemostatic efficacy will be assessed by the subject/caregiver and the investigator who will assess overall efficacy by a 4-point scale.

The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

The purpose of the study is to evaluate the efficacy and safety of BAX 855 in severe hemophilia A previously treated (PTP) males, 12 to 65 years of age who are undergoing elective surgical or other invasive procedures.

The main objectives of this study were to investigate the safety and efficacy of Replenine®-VF administered in appropriate dosage by bolus infusion to prevent bleeding and achieve haemostasis in subjects with haemophilia B undergoing surgery.