moderately severe to severe hemophilia B (FIX:C=2%) MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Sponsors
Pfizer Inc., 235 East 42nd Street, New York, NY 10017
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.Participant must be =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Anti-AAV-Spark100 nAb titer =1:1 (i.e., positive for nAb), at screening. 2.Participants who have current or prior history of inhibitor to FIX. 3.Known hypersensitivity to FIX replacement product or IV immunoglobulin administration. 4.History of thrombotic events including, but not limited to, stroke or myocardial infarction. 5.History of or any concurrent clinically significant major chronic disease, infection, or condition that the investigator deems unsuitable for participation. 6.ALT, AST, alkaline phosphatase >2 × ULN. 7.Bilirubin >1.5 × ULN (isolated bilirubin >1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin 0.48 is exclusionary), FibroScan (a score of >8 kPa is exclusionary), or shear wave elastography by conventional ultrasound (a score of >6.7 kPa is exclusionary). 15.Serological evidence of HIV-1 or HIV-2 with CD4+ cell count =200/mm3 or viral load >20 copies/mL. 16.Sensitivity to heparin or heparin-induced thrombocytopenia. 17.Sensitivity to the study intervention, or components thereof, or drug or other allergy that, in the opinion of the investigator or the sponsor's Medical Monitor, contraindicates participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate efficacy of a single infusion of PF-06838435 (fidanacogene elaparvovec) in male pediatric participants <18 years of age with moderately severe to severe hemophilia B (FIX:C=2%);Secondary Objective: •To further evaluate efficacy of a single infusion of PF-06838435 (fidanacogene elaparvovec) in male pediatric participants <18 years of age with moderately severe to severe hemophilia B (FIX:C=2%) •Safety and tolerability of a single infusion of PF-06838435 (fidanacogene elaparvovec) in male pediatric participants <18 years of age with moderately severe to severe hemophilia B (FIX:C=2%) •Assess durability of efficacy up to 5 years;Primary end point(s): •ABR for the first year post-PF-06838435 infusion versus ABR for prophylaxis FIX replacement regimen (retrospectively collected during the 1 year immediately prior to informed consent). •Steady-state vector-derived FIX:C level (from Week 12 to 1 year). ;Timepoint(s) of evaluation of this end point: from Week 12 to 1 year | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •AIR for the first year post-infusion Additional Secondary Endpoints: •Annualized dose and total factor consumption during the first year post infusion •Percentage of participants without bleeds during the first year post-infusion •Change from baseline in joint health as measured by the HJHS instrument at 1 year post-infusion •Change from baseline in quality of life measured by: Haem-A-QoL/Haemo-QoL Physical Health domain score at 1-year post-infusion, if age cohort appropriate •Change from baseline in activity level measured by pedHAL Function of the Legs domain score at 1-year post infusion, if age cohort appropriate •Incidence and severity of adverse events collected during the study;Timepoint(s) of evaluation of this end point: first year post infusion | — |
Countries
Canada, France, Germany, Italy, Japan, Korea, Republic of, Spain, Sweden, United Kingdom
Contacts
Public ContactClinical Trials.gov Call Centre
Pfizer Inc.
Outcome results
None listed