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Showing 1-20 of 79 trials
NCT03861273
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate \[ABR\] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).
NCT03961243
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
NCT06820515
The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems. Doctors, scientists, policymakers, and other health care providers need a large amount of information from a lot of people to answer scientific, public health, and policy questions about better ways to treat blood disorders. They will use the information from the ATHNdataset to answer these questions.
NCT06379789
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, both in the near term and over time. 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 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
NCT05932914
This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.
NCT05568459
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental treatment being tested in this study. The study is informational, and part of a larger program to understand and treat Hemophilia B with a potential experimental new therapy in the future. There is no obligation to agree to taking part in this future study. The study is looking to answer several other research questions to help understand each participant's individual disease characteristics, including: * How often to use FIX replacement therapy, both on a regular basis (prophylaxis) and as needed to treat bleeding events * Measurement of FIX activity (factor IX is a clotting factor) by different laboratories using different types of tests in Hemophilia B participants * Possible complications from the FIX replacement therapy the patient receives (usual standard of care will continue to be used) * How quality of life is affected by Hemophilia B * How joint health is affected by Hemophilia B * How often the participant visits the emergency room, urgent care center, physician's office, hospital, or has a telemedicine visit as a result of bleeding events * Whether the body makes antibodies (a protein produced by the body's immune system) against the FIX replacement therapy you receive, which could make the drug less effective or could lead to side effects
NCT06008938
This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
NCT04645199
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China. Method The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.
NCT07080905
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.
NCT06399289
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.
NCT06700096
The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.
NCT05044845
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low. Transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. Current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. In addition, few educational resources to explain basic gene therapy concepts exist in languages other than English. This study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries. Primary Objective: To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally Secondary Objectives: 1. To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally 2. To explore healthcare workers beliefs and attitudes about gene therapy 3. To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.
NCT06611436
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.
NCT06312475
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.
NCT06634836
The purpose of this study is to learn about experiences of patients with hemophilia A and B after taking gene therapy. The experiences of patients will be studied through online interviews. This study is seeking participants who are: * part of the Pfizer's gene therapy clinical studies or * in the long-term follow up for these clinical programs. Participants will have one study visit at the clinic and one online interview. The planned duration for each participant will be 1 to 2 months. This covers the time from entering the study to end of the online interview.
NCT05962398
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
NCT01154231
The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1. Occurrence status of adverse events 2. Factors that may influence the safety 3. Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.
NCT02740413
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
NCT05879549
Hemophilia is a bleeding disorder caused by a deficiency of clotting factors in the blood. Muscle and joint bleeding are frequently observed in patients with hemophilia. Lower extremity bleeding can adversely affect balance, gait, and proprioception. The goal of this study; To compare the effectiveness of closed kinetic chain exercises and proprioceptive exercises on balance, proprioception and gait parameters in adolescent and young hemophilic individuals with lower extremity joint involvement. The main question it aims to answer is: Are the effects of closed kinetic chain exercises and proprioceptive exercises different on balance and walking in hemophilic individuals?
NCT03938792
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.