View clinical trials related to Hemophilia A.
Filter by: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.
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
This observational study consists of two parts. In part one, case scenario focus groups with hemophilia A patients and healthcare providers (HCPs) will be held. This parts aims to identify potential use scenarios of a point of care (POC) in vitro medical for patients with hemophilia A. The main questions it aims to answer are: - How is coagulation lab testing for patients with hemophilia A currently organized? - What is the interest and what are desired alternatives of a POC in-vitro diagnostic medical device for patients with hemophilia A? Part two of the study consists of a use scenario study in which patients with hemophilia A and HCPs will evaluate two types of non-functional mock-ups of a POC in vitro medical device. The main goal of this part is to evaluate the usability of the current prototypes of the POC device.
This study is a single arm, open, multicenter clinical trial in which the previous treated severe hemophilia A patients receive recombinant human coagulation factor VIII for injection to evaluate the efficacy and safety of on-demand treatment in the event of new bleeding events. If evaluable surgical cases occur, the overall efficacy and safety of recombinant human coagulation factor VIII for injection as a replacement therapy for severe hemophilia A PTPs during the perioperative period will be evaluated.
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection.
The primary goal of this observational study is to determine if: - health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly different for boys receiving an intravenously administered factor replacement product compared to a subcutaneously administered non-factor replacement product, as measured at the 3 month time-point. - Burden of caring for a boy with moderate/severe hemophilia A is significantly different for parents/caregivers of boys with moderate/severe hemophilia A receiving an intravenously administered factor replacement product compared to a subcutaneously administered non-factor replacement product, as measured at the 3 month time-point. Persons with hemophilia and their caregivers will complete questionnaires at baseline, three months, six months, and one week after six months.
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.
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy. The recommended initial dose of prophylactic administration was 50 IU/kg, the dose range was 25 to 50 IU/kg, and the recommended frequency of administration was once every three days (Q3D). The dose range could be adjusted according to the patient's response. The main trial period was prophylaxis up to ≥50 exposure days (EDs) and ≥6 months. The investigator may adjust the dose according to the clinical efficacy of the subjects (the occurrence of bleeding and its clinical manifestations) and the concentration of FⅧ valley according to the following principles. If necessary, the investigator may adjust the dosing interval according to the clinical efficacy of the subject (the occurrence of bleeding and its clinical manifestations) and the concentration of FⅧ. Investigators are advised to inform sponsors or their research partners when adjusting doses and dosing intervals during prophylaxis. After participants completed prophylaxis until ≥50EDs and ≥6 months, participants' willingness and investigator evaluation were used to decide whether to enter the extended trial. All subjects entering the extended phase continued with the original prophylactic regimen until 100EDs was dosed. During the main trial period and the extended preventive treatment period, if the subjects have breakthrough bleeding events requiring treatment, hemostatic treatment of breakthrough bleeding with investigational drugs can be performed. The researchers can refer to the treatment guidance for different degrees of bleeding in Table 6-1. Taking into account the subject's prophylactic dose, severity of bleeding, site and extent of bleeding, clinical status, and previous PK results (if any), the investigator determines the appropriate dose to administer (recommended dose range: 25 to 50 IU/kg) and dosing times until the investigator assessed significant control of bleeding episodes (e.g. reduction of pain and swelling) or return to pre-bleeding activity. If the bleeding episode stops, the subject will continue with the same dose and frequency of prophylactic medication as before the bleeding episode.
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.
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.