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Hemophilia B clinical trials

View clinical trials related to Hemophilia B.

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NCT ID: NCT06399289 Not yet recruiting - Hemophilia B Clinical Trials

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Start date: June 2024
Phase: Phase 3
Study type: Interventional

This study will investigate the pharmacokinetics (PK), efficacy, and safety of rIX-FP for the routine prophylaxis of bleeding episodes in male Chinese previously treated patients (PTPs) with hemophilia B (FIX activity of ≤ 2%). In addition to the scheduled rIX-FP prophylaxis regimen, subjects may also receive rIX-FP episodic (on-demand) treatment for breakthrough bleeding episodes and rIX-FP for the prophylaxis and treatment of bleeding in emergency surgical procedures.

NCT ID: NCT06379789 Not yet recruiting - Hemophilia B Clinical Trials

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B

BEYOND-9
Start date: July 25, 2024
Phase: Phase 1/Phase 2
Study type: Interventional

Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the "study drug". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy. The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it. The study is looking at several other research questions including: - How much study drug is in the blood at different times - Whether the body makes antibodies against parts of the study drug, which could make the drug less effective or could lead to side effects. Antibodies are proteins produced by the body's immune system in response to a foreign substance - Whether the body makes antibodies against the clotting factor replacement therapy - How quality of life is affected by hemophilia B and if it changes after taking study drug - How joint health is affected by hemophilia B and if it changes after taking study drug - How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug - How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug) - Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood

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: 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: NCT06285071 Enrolling by invitation - Haemophilia A Clinical Trials

Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors

Start date: April 30, 2024
Phase:
Study type: Observational

The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.

NCT ID: NCT06158334 Recruiting - Hemophilia A Clinical Trials

Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®)

CHALE
Start date: June 1, 2022
Phase:
Study type: Observational

Haemophilia A and haemophilia B are inherited bleeding disorders resulting from the absence or deficiency of coagulation factors VIII and IX, respectively. The peri-operative period of people with haemophilia is commonly managed with replacement therapy. In phase 3 studies of Elocta® (extended half-life recombinant factor VIII-Fc) and Alprolix® (extended half-life recombinant factor IX-Fc), haemostatic efficacy was demonstrated to be good or excellent, close to the haemostatic efficacy usually seen in people without haemophilia, with maintenance and stability of circulating FVIII and FIX levels compatible with the surgical procedure, while reducing the frequency of infusions and consumption of therapeutic units. In 2019, the National Protocol for Diagnosis and Care in haemophilia recommended 2 methods for managing patients with haemophilia in the peri-operative period, either discontinuous injections of standard or extended half-life factor VIII/IX or a continuous infusion of FVIII/IX. Many countries, including France, have adopted these rFVIII/IXFc therapeuitic products and recommended their use in the surgical management of patients. However, the use of these two products in real life during surgery in haemophilic A and B patients has not been described in detail. It seems therefore relevant to better document their use in order to progressively specify their use during surgeries with varied bleeding risks.

NCT ID: NCT06147414 Not yet recruiting - Cystic Fibrosis Clinical Trials

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

DANNIgene
Start date: April 2024
Phase:
Study type: Observational

Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.

NCT ID: NCT06125756 Not yet recruiting - Clinical trials for Hemophilia B Without Inhibitor

Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

Start date: December 20, 2023
Phase: Early Phase 1
Study type: Interventional

This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.

NCT ID: NCT06120582 Recruiting - Hemophilia B Clinical Trials

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Start date: May 2, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.

NCT ID: NCT06119659 Not yet recruiting - Clinical trials for Hemophilia B Without Inhibitor

Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

Start date: November 20, 2023
Phase: Early Phase 1
Study type: Interventional

This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.