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Hemophilia A clinical trials

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NCT ID: NCT06461533 Recruiting - Clinical trials for Acquired Hemophilia A

A Survey of Susoctocog Alfa (Genetical Recombination) in Participants With Acquired Haemophilia A

Start date: June 10, 2024
Phase:
Study type: Observational

This study is a survey in Japan of Susoctocog Alfa (Genetical Recombination) intravenous injection used to treat participants with bleeding events of acquired Haemophilia A (AHA). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from Susoctocog Alfa (Genetical Recombination) intravenous injection and to check if Susoctocog Alfa (Genetical Recombination) intravenous injection improves bleeding events of AHA. During the study, participants with AHA will take Susoctocog Alfa (Genetical Recombination) intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from Susoctocog Alfa (Genetical Recombination) intravenous injection for up to 90 days after the last dose of study drug or until discontinued (varied from participant to participant).

NCT ID: NCT06352216 Recruiting - Haemophilia A Clinical Trials

Prevalence of Synovitis in Patients With Haemophilia A

SynoPrev
Start date: April 15, 2024
Phase:
Study type: Observational

The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.

NCT ID: NCT06349473 Recruiting - Clinical trials for Healthy Participants

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B)

Start date: April 15, 2024
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604 in participants with severe Hemophilia A or Hemophilia B, with or without inhibitors (Part B).

NCT ID: NCT06320626 Recruiting - Child Clinical Trials

Pharmacokinetic-guided Dosing of Emicizumab

DosEmi
Start date: September 8, 2022
Phase: Phase 4
Study type: Interventional

The goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of bleeding in congenital haemophilia A patients.

NCT ID: NCT06312475 Recruiting - Clinical trials for Hemophilia A With Inhibitor

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Start date: January 9, 2024
Phase: Phase 3
Study type: Interventional

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use. Successfully screened participants will be randomly assigned to KN057 Prophylaxis (Arm 1) versus No Prophylaxis (Arm 2) at a ratio of 2:1. Participants in KN057 Prophylaxis will receive KN057 prophylaxis for 52 weeks upon enrollment. Participants in No Prophylaxis will first receive on-demand treatment for 26 weeks, then switch to KN057 prophylaxis for 26 weeks.The trial period is 59 weeks, including a 3-week screening period, a 26-week main trial, a 26-week extension period, and a 4-week follow-up period after the last administration.

NCT ID: NCT06297486 Recruiting - Hemophilia A Clinical Trials

Study of a Gene Therapy Treatment for Hemophilia A

KEYSTONE 1
Start date: March 13, 2024
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.

NCT ID: NCT06289166 Recruiting - Hemophilia Clinical Trials

Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Start date: March 18, 2024
Phase: Phase 2
Study type: Interventional

This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.

NCT ID: NCT06273865 Recruiting - Hemophilia A Clinical Trials

Version Testing of EnzySystem Version A for Hemophilia A

Start date: May 29, 2024
Phase:
Study type: Observational

Background of the study: To measure blood clotting, blood is taken from a vein. This blood is processed in the laboratory and then tested. A new device has been developed that requires only a very small volume of blood (5-10 drops of blood) to perform the laboratory tests. The long-term goal is that this device can be used by a doctor or at home to quickly measure blood clotting. In this research we want to compare this new system with the standard methods - measurement in the laboratory - and evaluate whether the correct value is determined. Objective of the study: The primary objective of this study is to demonstrate that the EnzySystem HemA version A can record thrombin generation TG and quantify FVIII activity levels within a time frame of 60 min in fresh whole blood samples of healthy volunteers and patients with hemophilia A in the Enzyre laboratory (for healthy volunteers) and the Radboudumc (for patients with hemophilia A). Study design: This is a cross-sectional observational study. All participants are asked to fill a questionnaire prior to blood collection. The blood of healthy volunteers will be collected in an office of Enzyre BV, the blood of patients will be collected in the Radboudumc. Blood collection, by venepuncture, will be conducted by a Radboudumc research nurse or physician of the research team in both locations. In total, four blood tubes with citrate as anticoagulant will be drawn (a total of around 11 mL). Study population: The study population consists of 20 healthy volunteers: evenly distributed between male and female; ages spread over the range from 20 to 70 years old; recruited by Enzyre via advertisement. 20 Patients: 5 severe hemophilia A; 5 moderate hemophilia A; 10 mild hemophilia A; recruited from the Hemophilia Treatment Center (HTC) Nijmegen-Eindhoven-Maastricht (NEM) (location Radboudumc). Primary study parameters/outcome of the study: Demonstrate that the EnzySystem HemA version A can record TG and quantitative FVIII activity levels within a time frame of 60 min in fresh blood samples of healthy volunteers and patients with hemophilia A. Secondary study parameters/outcome of the study (if applicable): Secondary study parameters are composed whether the measured values comply with the desired assay specificity and accuracy. Outcomes are analysed for equivalence compared to one-stage FVIII assay, FVIII chromogenic assay, thrombin generation via the Nijmegen Hemostasis Assay, and possibly via the Technoclone assay. Is it possible to measure FVIII activity with the EnzySystem HemA version A in fresh blood samples of healthy volunteers and patients with hemophilia A, compared to the gold standard with; - Precision in the normal range (60-140%): min. 30% - Precision in the low range (3-10%): min. 50% - Limit of Detection range min. 100 % FVIII activity - Limit of Detection low range min. 3 % FVIII activity Is it possible to measure TG with the EnzySystem HemA version A in fresh blood samples of healthy volunteers and patients with hemophilia A, compared to the gold standard with; - Precision in the normal range (60-140%) of control samples: min. 30% - Precision in patient with hemophilia A: min. 50% - Limit of Detection, high range > 400 nM thrombin activity - Limit of Detection measured with Plasma, low range < 50 nM thrombin Other study parameters All samples will also be tested for other hemostasis specific parameters as these parameters may affect a proper measurement of both FVIII activity and Thrombin Generation. The following parameters will be measured in plasma obtained from the whole blood vacutainers. Moreover, left over samples (plasma) will eventually be used to develop other coagulation related parameters. - von Willebrand Factor antigen levels - von Willebrand Factor ristocetin activity levels - Prothrombin Fragment 1+2 levels - ADAMTS13 activity - FVIII antigen levels - blood group

NCT ID: NCT06267209 Recruiting - Hemophilia Clinical Trials

Conditioned Pain Modulation in Patients With Hemophilia

Start date: May 1, 2024
Phase:
Study type: Observational

Introduction: Hemophilic arthropathy is characterized by functional alterations, disabling physical sequelae, and chronic pain. Conditioned pain modulation describes the net effect of endogenous pathways that enhance or diminish the effects of afferent noxious stimuli. Objectives: To describe conditioned pain modulation in patients with hemophilia and identify the best predictive model of conditioned pain modulation in these patients Methods: Cross-sectional cohort study. 51 patients with hemophilic arthropathy will be recruited in 3 regions of Spain. The main study variable will be the conditional pain modulation (Conditioned Pain Modulation Index, using an ischemic technique of the arm using the pain pressure threshold as a test stimulus), with age being the dependent variable. The secondary variables, estimated as modifying or confounding variables, will be kinesiophobia (Tampa Scale for Kinesiophobia), catastrophizing (Pain Catstrophizing Scale), trait and state anxiety (State-Trait Anxiety Inventory) and the main clinical, anthropometric, and sociodemographic. Expected results: Identify the degree of modulation conditioned by pain in patients with hemophilic arthropathy. Identify the best predictive model for conditioned pain modulation in these patients based on the study variables

NCT ID: NCT06261593 Recruiting - Hemophilia Clinical Trials

Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy

Start date: April 6, 2024
Phase: N/A
Study type: Interventional

Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy). This sequela is manifested by chronic pain, limited range of motion, axial abnormalities, and periarticular muscle atrophy. Objective. To assess the safety and effectiveness of an intervention through blood flow restriction, regarding the frequency of bleeding and the improvement in muscle activation and strength, range of motion, stability, joint pain, joint status and the perception of quality of life in patients with hemophilic ankle arthropathy. Study design. Randomized, multicenter, single-blind clinical study. Method. 32 patients with hemophilia A and B will be recruited in this study. Patients will be recruited in 4 regions of Spain. The dependent variables will be: bleeding frequency (self-registration), pain (measured with the visual analog scale and pressure algometer), quality of life (SF-36 scale), joint status (Hemophilia Joint Health Score scale), strength (dynamometer) and muscle activation (surface electromyograph), range of motion (goniometer) and stability (The Single Leg Stance Test). Three evaluations will be carried out: pre-treatment, post-treatment and after a follow-up period of 4 weeks. Expected results. Observe the safety of blood flow restriction in hemophilia patients. To analyze the efficacy of blood flow restriction in improving muscle strength and activation, range of motion, chronic pain, stabilit, and the perception of quality of life in patients with hemophilic ankle arthropathy.