Hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Product Name: giroctocogene fitelparvovec
Product Code: [PF-07055480]
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: giroctocogene fitelparvovec
Current Sponsor code: PF-070
Sponsors
PFIZER INC
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Males who completed 6 months of routine Factor VIII prophylaxis therapy during the lead in study(C0371004) and have = 150 documented exposure days to a Factor VIII protein product • Moderately severe to severe hemophilia A (Factor VIII activity = 1%) • Suspension of FVIII prophylaxis therapy post study drug infusion For full list of inclusion criteria please refer to study protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 63 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Anti-AAV6 neutralizing antibodies • History of inhibitor to Factor VIII • Laboratory values at screening visit that are abnormal or outside acceptable study limits • Significant and/or unstable liver disease, biliary disease, significant liver fibrosis • Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit • Active hepatitis B or C •Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count =200 mm3 and/or viral load >20 copies/mL For full list of exclusion criteria please refer to study protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the efficacy of a single infusion of PF-07055480 in participants =18 and <65 years of age with moderately severe to severe hemophilia A (FVIII C = 1%).;Secondary Objective: - To demonstrate that the use of exogenous Factor VIII (FVIII) is significantly reduced post PF-07055480 infusion. - To compare additional efficacy parameters post PF-07055480 infusion including use of exogenous FVIII, information on bleeding events and patient-reported outcomes (PROs). - Estimate the durability of efficacy up to 5 years after PF-07055480 infusion. - To estimate the safety and tolerability of PF-07055480, including immunogenicity, for the study duration of 5 years after PF-07055480 infusion.;Primary end point(s): Annualized bleeding rate (ABR, spontaneous and traumatic bleedings) through 12 months following PF-07055480 infusion (from Week 3) versus ABR on prior Factor VIII (FVIII) prophylaxis replacement regimen.;Timepoint(s) of evaluation of this end point: From Week 3 to 12 months (primary analysis) post study drug infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Factor VIII (FVIII) activity level after the onset of steady state and through 12 months following infusion of PF-07055480. - Annualized infusion rate (AIR) of exogenous FVIII through 12 months following infusion of PF-07055480 versus AIR on prior FVIII prophylaxis replacement regimen. - The following secondary parameters will be assessed through 12 months after PF-07055480 infusion and compared with prior FVIII prophylaxis replacement regimen: • Annualized FVIII consumption. • Annualized bleeding rate (ABR) of specific type (from Week 3): o by cause (spontaneous or traumatic) o by location (in joints, in target joints, or in soft tissue). • Total ABR (treated and untreated) from Week 3. • Total ABR by location (in joints, in target joints, or in soft tissue) from Week 3. • Percentage of participants without bleeds. • Change in joint health as measured by the Hemophilia Joint Health Score (HJHS) instrument. • Change from baseline at 12 months in the following patient-reported outcome (PRO) endpoints: o Haemophilia Quality of Life Questionnaire for Adults (Haem-AQoL) o Haemophilia Activities List (HAL). - The following parameters will be analysed yearly and throughout the 5-year study period: • ABR. • Steady state FVIII activity level. • AIR of exogenous FVIII. • Annualized FVIII consumption. • ABR of specific type: o by cause (spontaneous or traumatic). o by location (in joint, in target joints, or in soft tissue). • Total ABR (treated and untreated). • Total ABR by location (in joint, in target joints, or in soft tissue). • Percentage of participants without bleeds. • Change in joint health as measured by the HJHS instrument. • Change in PRO endpoints: Haem-A-QoL and HAL. - Incidence and severity of adverse events (AEs). - Events of special interest (such as hypersensitivity reactions, clinically reported thrombotic events, and malignancy). - Immunogenicity: • Antibodies against adeno-associated virus 6 (AAV6) capsid protein (neutralizing | — |
Countries
Australia, Belgium, Brazil, Canada, France, Germany, Greece, Israel, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trials.gov Call Centre
Pfizer Inc.
Outcome results
None listed