Active clinical trials for "Hemophilia A"

This is a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.

Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough bleeding, number of hospital stay and day-off from school? Type Research: Clinical research Study design: Single center clinical trials Concise methodology: Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study. Observation and measurement: History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF) Measurement: i. Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients Potential impacts: The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality of life. The result of this study will guide further study on optimal dose and duration of factor VIII treatment of hemophilia A patients in the future.

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

Severe hemophilia is characterized by frequent and lifelong bleeding, with more than 60% of bleeds occurring into joints . Repeated joint bleeding leads to chronic synovitis, cartilage damage and bony destruction. Currently available treatment of hemophilic arthropathy, such as analgesics, NSAIDs, and hyaluronic acid (HA), are predominantly directed toward the symptomatic relief of pain and inflammation, but they do little to reduce joint cartilage degeneration. Platelet-Rich Plasma (PRP) is a simple and minimally invasive method that provides a natural concentrate of autologous growth factors from the blood. This method is now being increasingly applied in clinical practice to treat musculoskeletal disorders, such as tendon repairment and osteoarthritis. To the best of our knowledge, no study applies PRP for arthropathy of knee joint in hemophilia patients. The aim of the study is to investigate the efficacy, safety and duration of benefit of single PRP injection versus five weekly intra-articular injections of HA in patients with hemophilic arthropathy of knee.

This trial is conducted in Africa, Asia, Europe and North America. The aim of the trial is to assess the efficacy of concizumab administered s.c. (subcutaneously, under the skin) once daily in preventing bleeding episodes in haemophilia A and B patients with inhibitors.

This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W) and once every 4 weeks (Q4W) regimens in pediatric participants.

The purpose of this study is to: 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min 2. To evaluate the tolerability and safety of infusing reduced volume FEIBA at increased rates of 4 and 10 U/kg/min, in comparison to the standard rate of 2 U/kg/min at the regular volume

Although arthropathy is a serious problem in patients with hemophilia due to the associated morbidity and incapacity, to the best of the investigators knowledge, no studies have looked at the effect of educational physiotherapy for its clinical improvement. This contribution presents the results of educational physiotherapy program applied for 15 weeks with home exercises - in patients with hemophilic arthropathy. After treatment, experimental group showed improved a significant reduction of pain, and best quality of life al illness behaviour. During treatment no patient showed elbow haemarthrosis, which underlines the safety of this physiotherapy program.

The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.

Comparison of prophylaxis and on-demand treatment in children with moderate to severe hemophilia A. This study determines to compare the efficacy of prophylaxis and on-demand treatment in moderate to severe hemophilia A children in King Chulalongkorn Memorial Hospital, Bangkok, Thailand