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Found 6 clinical trials
A Phase 1/2 Study of SHP648 an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.

christmas disease
viral vector
hemophilia
gene therapy
prophylactic
  • 0 views
  • 19 Feb, 2024
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objective: To characterize the safety and tolerability To characterize the pharmacokinetics (PK)

hemophilia
fitusiran
haemophilia a
antihemophilic factor
factor ix
  • 0 views
  • 19 Feb, 2024
  • 11 locations
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX

Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes Spontaneous bleeding episodes Target joint bleeding episodes To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants …

hemophilia
fitusiran
haemophilia a
antihemophilic factor
hemarthrosis
  • 0 views
  • 19 Feb, 2024
  • 41 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objective: To characterize the safety and tolerability To characterize the pharmacokinetics (PK)

hemophilia
fitusiran
haemophilia a
antihemophilic factor
factor ix
  • 0 views
  • 19 Feb, 2024
  • 6 locations
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.

antihemophilic factor
hemophilia
  • 0 views
  • 19 Feb, 2024
Pharmacokinetics Pharmacodynamics and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to …

acute respiratory syndrome (sars)
SARS
  • 0 views
  • 19 Feb, 2024