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We list hundreds of Clinical Trials about "Chugai Pharma Emicizumab Receives Priority Review Designation Hemophilia" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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This randomized, multicenter, open-label, Phase 3 study is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII (FVIII) inhibitor status. Participants who received episodic therapy with FVIII or bypassing agents prior to study entry and experienced at least 5 bleeds over the prior 24 weeks will be randomized in a 2:2:1 ratio to the following regimens: 1) Emicizumab prophylaxis at 3 milligram...
This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks in participants with hemophilia A with or without inhibitors against factor VIII (FVIII). The study consists of 2 parts: a pharmacokinetic (PK) run-in part followed by an expansion part.
This Phase IV, multicenter study will evaluate whether participants with Hemophilia A and inhibitors receiving emicizumab prophylaxis can safely undergo minor surgical procedures without prophylactic bypassing agents (BPA).
This single-arm study will enroll children (less than [
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Approximately 200 participants, aged 12 or older, will be enrolled in this study and are expected to be enrolled at approximately 85 sites globally. Participants will receive an initial weekly dose of prophylactic emicizumab subcutaneously for ...
This is a randomized, multicenter, open-label, Phase 3 clinical study in participants aged 12 years or older to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in participants with severe hemophilia A without inhibitors against FVIII.
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate. Physicians complete a retrospective chart review on each enrolled patient. Patients will complete a one-time study questionnaire.
This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.
To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two established hemophilia mutation testing laboratories (one in Europe and one in North America).
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
The purpose of this post-marketing safety study is to evaluate the safety, immunogenicity, and effectiveness of ADVATE in previously untreated patients (PUPs) in China with moderate to severe hemophilia A.
To correlate the HLA type and genetic defect with hemophilia A.
To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.
Prenancy and specialy delivery for hemophilia mother and her children possibly hemophilia is risked. Diagnostical of hemophilia pregnan is the first difficulty but patients with hemorrhagic menstruation without etiology known, hemorrhagic complication during a chirurgical procedure or during previously pregnancy currently are examine in Haemostasis consultation. None French recommendation specifie for health practice of these hemophilia patients exists. However, Anglo-S...
To collect and analyze data on female carriers of severe and moderate hemophilia A and B.
To assess efficacy and safety of BeneFix® for prophylaxis in "Short-term" therapy and on demand therapy for all bleeding episodes of subjects with hemophilia B.
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder. The study is designed to determine whether there are problems and issues unique to females with hemophilia.
This study is a Phase I/II trial using a novel bioengineered lenti viral vector to deliver a normal gene for human clotting factor VIII or factor IX into patients with hemophilia A or hemophilia B, to evaluate the safety, efficacy and pharmacokinetics of IV infusion of gene modified autoHST in patients.
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient data and guide clinical practice. With informed consent from the patient, the WBDR stores anonymous data about the person's disease, such as hemophilia type and severity, symptoms, and treatment.
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in participant-identified goal areas in individuals with hemophilia A.
Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding. Bleeding is prevented and/or treated with recombinant factor VIII (rFVIII), which is an FDA-approved treatment for Hemophilia A. Obesity is common among patients with hemophilia. Some studies have shown that obese hemophilia patients may be able to prevent bleeding with a ...
The Swiss Hemophilia Registry will collect data on the prophylactic and therapeutic use of factor concentrates in patients with hemophilia and other severe bleeding disorders in Switzerland.
Spanish Validation of HAL and HEP questionnaire by double translation (English-Spanish-English), with a pilotage pediatric patients with hemophilia and adults and parents of children with hemophilia. The final validity will be obtained with a sample of 60-100 patients with hemophilia