Active clinical trials for "Hemophilia A"

This study will be used to assess the feasibility and sensitivity of using the iSTEP, to assess exercise capacity in boys with haemophilia. The feasibility criteria to be explored includes: Recruitment to target number or better The test procedure is completed within an allocated time (1-2 hours) and by 90% of participants without serious adverse events 90% of participants achieve a sufficient exercise response (85% maximum heart rate (HRmax)) Calculation of estimates of minimum clinically important differences and variability for sample size calculations and responsiveness to severity of haemophilia and orthopaedic status The iSTEP exercise test will be compared to a more commonly used exercise test the modified shuttle walk test (10m- MSWT). Muscle strength (using myometry) will also be tested and compared to exercise performance for any relationships. Physical activity levels will also be assessed to obtain some baseline measurements of physical activity levels in this patient population, which are currently not very well known. The overarching aim of this current study is also to establish a robust and sensitive exercise test and to gain an understanding of the effects of physical activity levels and muscle strength on exercise capacity in this population.

This study aims to assess the effects of progressive resistance training compared with aerobic training, among those with hemophilia. Participants will be divided into two groups. The control group will receive an aerobic exercise protocol, and the resistance group will follow a resistance training program. Each group will receive a total of eight sessions. Target muscles are deltoids, biceps, triceps, quadriceps, hamstrings, and calves. Subjective and objective assessment will be performed for each group, to determine the effectiveness of each intervention.

This Phase 1 study will be a single-arm, open-label, non-randomized, non-controlled investigation of the safety, tolerability, pharmacokinetics, and pharmacodynamics of PF-06741086 in Chinese adult participants with severe hemophilia.

Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.

Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to control of hemorrhaging in both the prophylaxis and on demand groups of IB1001 in subjects with hemophilia B. Key Secondary Objectives: To evaluate the ability of IB1001 to provide coverage against bleeding under surgical circumstances; To evaluate the long-term safety and efficacy of IB1001

The study will see if counselling adolescents with severe or moderate Hemophilia A or B results in increased compliance in the maintenance of bleeding logs.

The purpose of the study is to compare the effects of recombinant factor VIIa (NovoSeven) on restoring coagulation activation between patients with hemophilia A and antibodies to factor VIII and normal subjects (controls) by use of an in vivo method.

In persons with severe haemophilia A infused factor VIII half-life and other pharmacokinetic can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age. However, FVIII pharmacokinetics (PK) has not been throughly studied in patients with severe HA as a function of daily physical activity. It si known that vigorous intensity physical activity/exercise can transiently but significantly increase circulating levels of endogenous VWF and consequently FVIII in normal subjects and in patients with moderate or mild haemophilia A. The proposed study is a Proof of Concept one as it will be aimed at investigating the relation between daily physical activity, measured by Sensewear armband device, as number of daily steps and PK variability of infused rec-FVIII concentrate. This kind of investigation has never been done and it is a great interest also for the evaluation of patient's qualitiy of life.

Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions delivered as part of a patient education package. The study will capture severe haemophilia A patient reported outcome measures before and after PK-focused dosing discussions, including a standardised patient education package, that include personalised PK-guided dosing suggestions from a computational predictive device (myPKFiT®). The pragmatic study design recognises the CE marked myPKFiT® device is being implemented into routine care nationally and consequently only requires a single additional clinic visit for the purpose of consenting.

This study hypothesizes that the study drug, PF-05280602 (at the selected doses) will be safe to administer to subjects with severe Hemophilia A or B with or without inhibitors and will demonstrate evidence of hemostatic activity. This is supported by the preclinical findings in hemophilic animal models.