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Found 24 clinical trials
Evaluation of a Recombinant Factor IX Product APVO101 in Previously-Treated Pediatric Patients With Hemophilia B

Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.

factor ix activity
blood components
nonacog alfa
hemophilia
renal function
  • 8 views
  • 25 Jan, 2021
  • 5 locations
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

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

factor ix activity
gene therapy
hemophilia
  • 37 views
  • 22 Jun, 2021
  • 44 locations
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B

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 …

acute bleeding
acute hemorrhage
Factor VIII
antihemophilic factor
replacement therapy
  • 58 views
  • 14 Jul, 2021
  • 75 locations
Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C 2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C 1%)

To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish a minimum of 6 months of prospective …

gene therapy
Factor VIII
antihemophilic factor
severe haemophilia a
replacement therapy
  • 210 views
  • 02 Aug, 2021
  • 89 locations
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

factor ix
blood components
coagulation factor
nonacog beta pegol
hemophilia
  • 379 views
  • 15 Mar, 2021
  • 75 locations
Personalized Medicine for Canadians With Hemophilia

Performing an individual pharmacokinetic (PK) estimate is only the first step in implementing tailored prophylaxis, which requires using the PK profile information to design a personalized treatment regimen matching the treatment needs of individual patients. The overarching goal of WAPPS-Hemo is to provide an easy-to-use web application supporting all the …

treatment regimen
severe haemophilia a
hemophilia
  • 2 views
  • 24 Jan, 2021
  • 6 locations
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients

alternative factor IX treatments in Taiwan.

coagulation factor
christmas disease
factor ix
  • 0 views
  • 26 Jan, 2021
  • 1 location
Gene Therapy for Chinese Hemophilia B

hemophilia B subjects with 2IU/dl residual FIX levels. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of

gene therapy
arthropathy
coagulant
hemophilia
anaphylaxis
  • 0 views
  • 25 Jan, 2021
  • 1 location
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation substudy evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up.

hemophilia
  • 96 views
  • 22 Jul, 2021
  • 10 locations
Haemophilia and Bone Loss - PHILEOS Study

Haemophilia is a rare bleeding disorder, characterized by factor VIII (HA) or factor IX (HB) deficiency. The absence or the reduction of fVIII or fIX result in impaired thrombin generation and

hepatitis
hemarthrosis
bleeding tendency
clot formation
osteoporosis
  • 15 views
  • 11 Apr, 2021
  • 19 locations