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Hemophilia A clinical trials

View clinical trials related to Hemophilia A.

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NCT ID: NCT04076306 Completed - Hemophilia Clinical Trials

What is the Feasibility of the ISTEP Exercise Test in Boys With Haemophilia

Start date: January 25, 2018
Phase: N/A
Study type: Interventional

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: 1. Recruitment to target number or better 2. The test procedure is completed within an allocated time (1-2 hours) and by 90% of participants without serious adverse events 3. 90% of participants achieve a sufficient exercise response (85% maximum heart rate (HRmax)) 4. 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.

NCT ID: NCT04072237 Completed - Hemophilia A Clinical Trials

Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia

Start date: September 24, 2019
Phase: Phase 1
Study type: Interventional

This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor.

NCT ID: NCT04055051 Completed - Clinical trials for Liver Transplantation

ATHN 11: Liver Transplantation Outcomes Study

HOT
Start date: June 18, 2020
Phase:
Study type: Observational

This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.

NCT ID: NCT04015492 Completed - Hemophilia A Clinical Trials

Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII)

Start date: August 8, 2019
Phase: Phase 1
Study type: Interventional

This study is being conducted to compare how the body distributes and excretes the drugs Jivi (BAY 94-9027) and Adynovi. Jivi is a recently approved blood clotting Factor VIII (FVIII) medication for the treatment of hemophilia A (bleeding disorder resulting from a lack of FVIII). Both drugs are FVIII products which have been manufactured via recombinant technology and have an extended half-live, i.e. they will stay longer in the body than other FVIII products. Therefore these products act longer in the body which reduces the frequency of drug injections. To compare the two drugs, a cross-over design was chosen, i.e. each patient will receive both products one after another. Patients participating in this study will receive one dose of Jivi and one dose of Adynovi. Both drugs are injected into a vein. Observation will last for about 10 weeks, and blood samples will be taken from the participants to measure the blood levels of FVIII. Generic name of Jivi is Damoctocog-alfa-pegol, generic name of Adynovi is Rurioctocog alfa pegol.

NCT ID: NCT03995784 Completed - Hemophilia B Clinical Trials

Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

Start date: June 18, 2019
Phase: Phase 2
Study type: Interventional

Phase 2b, single-center, open-label study designed to evaluate the pharmacokinetics, pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens with CB2679d in 6 adult, male subjects with severe congenital hemophilia B.

NCT ID: NCT03951103 Completed - Clinical trials for Hemophilia A With Inhibitor

rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

Start date: November 7, 2018
Phase:
Study type: Observational

A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.

NCT ID: NCT03935334 Completed - Clinical trials for Hemophilia A or B With Inhibitor

Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors

Start date: July 23, 2018
Phase: Phase 3
Study type: Interventional

The purpose of this multicentre, randomized, double-blinded, single dose, two-way cross-over study, is to compare the pharmacokinetics (PK) and pharmacodynamic (PD) of two different doses of the biosimilar eptacog alfa (activated) with Novoseven in 48 patients, adult and children (>12 years), not bleeding, with hemophilia A or B with inhibitors. Patients will be randomized to receive either a single dose of eptacog alfa biosimilar 90 μg/kg or 270 μg/kg and one single dose of NovoSeven 90 μg/kg or 270 μg/kg, or vice versa, with doses separated by a washout period. All patients will be followed 12 months and will receive biosimilar eptacog alfa, on demand, for every bleeding episode that should occur - or - for prophylaxis, with the aim of monitoring of inhibiting antibody formation, lack of efficacy and collection of safety data.

NCT ID: NCT03915080 Completed - Hemophilia A Clinical Trials

Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

myPKFiT
Start date: December 2016
Phase: Phase 4
Study type: Interventional

MyPKFiT is a web-based application recently developed by Baxalta for the use in patients treated with Advate. MyPkFit has its basis in Bayesian forecasting, which allows estimation of individual PK parameters by a sparse sampling schedule, where only 2-3 samples are taken between 4 and 48 hours post infusion. With myPKFiT, it will, therefore, be possible to define an individual PK curve for each patient based on just a few sampling points and hence, taking the bleeding phenotype and the life style into account, potentially adjust the prophylactic treatment accordingly to optimize cost-effectiveness.

NCT ID: NCT03914716 Completed - Hemophilia Clinical Trials

Imaging of Arthropathy in Boys With Hemophilia in China

Start date: March 21, 2018
Phase: N/A
Study type: Interventional

Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA). It is thought that clinical and imaging manifestations of HA are at least partially attributable to genetic polymorphisms unrelated to the hemophilia genotype. Identifying and characterizing biologic factors that could explain differences in susceptibility to joint degeneration of patients with hemophilia would help stratify patients according to the risk of degeneration of their joints and develop personalized therapeutic and prophylactic strategies. This study is conducted in China.

NCT ID: NCT03876301 Completed - Hemophilia A Clinical Trials

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

Start date: January 21, 2019
Phase:
Study type: Observational

The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.