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NCT Number: NCT05568719

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia B Hemorrhage Hemorrhagic Disorders Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

-Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.

Exclusion criteria

-None

Treatment and study plan

Testing of hepatic AAV Vector integration

Diagnostic Test

Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated

Primary outcomes

  1. Incidence of thromboembolic events

    Time frame: Day 1 to 10 years

  2. Incidence of factor inhibitor development

    Time frame: Day 1 to 10 years

    FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL).

  3. Incidence of hepatic malignancy

    Time frame: Day 1 to 10 years

  4. Incidence of liver abnormalities

    Time frame: Day 1 to 10 years

  5. Factor activity level

    Time frame: Day 1 to 10 years

    Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.

Secondary outcomes

  1. Total ABR (treated or untreated; (excluding bleeds related to surgery)

    Time frame: Day 1 to 10 years

    ABR (Annual Bleed Rate): number of bleeding episodes per year. This includes treated and untreated bleeds.

    The ABR or the annualized number of bleeding episodes per year, will be derived for each participant for each observation period by using the following formula:

    ABR = (Number of bleeds / Days in observation period) x 365.25 days/year.

  2. Incidence of and time from vector infusion to resumption of prophylaxis

    Time frame: Day 1 to 10 years

    Describe incidence of resumption of prophylaxis resumption and the time (in days) to resumption of prophylaxis after receiving vector infusion.

  3. AIR of exogenous factor (excluding infusions related to surgery)

    Time frame: Day 1 to 10 years

    The AIR or the annualized number of FIX infusions per year, will be derived for each participant for each observation period by using the following formula:

    AIR = (Number of FIX infusions / Days in observation period) x 365.25 days/year.

  4. Consumption of exogenous factor (excluding infusions related to surgery)

    Time frame: Day 1 to 10 years

    The annualized TFC in international units (IU) will be derived for each participant for each observation period using the following formula:

    Annualized TFC = (Total units of FIX infused (IU)/ Days in observation period) x 365.25 days/year

  5. Incidence of Non-hepatic malignancy

    Time frame: Day 1 to 10 years

  6. Incidence of Auto-immune disorders

    Time frame: Day 1 to 10 years

  7. Incidence of SAEs

    Time frame: Day 1 to 10 years

    An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; development of a clinical thrombotic event; development of factor inhibitor; development of a hepatic malignancy; development of drug-related elevated hepatic transaminases that fail to improve with immunosuppressive regimens; occurrence of a malignancy with reasonable possibility of being related to study drug.

  8. All cause mortality

    Time frame: Day 1 to 10 years

    All-cause mortality was defined as the death due to any cause during the course of study. Incidence rate was defined as the total number of participants with admissible events divided by the total (for all qualifying participants) time at risk for the cohort/treatment group of interest. Incidence rate of all-cause deaths was reported in this outcome measure.

  9. EQ-5D-5L dimension and VAS scores

    Time frame: Day 1 to 10 years

    The EQ-5D-5L comprises a 5-item health status measure and a visual analog rating scale/feeling thermometer. Using the 5-dimensional Health State Classification, participants are asked to respond to five questions on different aspects of their health status that assess the following:

    • Mobility
    • Self-care
    • Usual activities
    • Pain/Discomfort
    • Anxiety/Depression

Study contacts

Contact information is provided by the study sponsor or research team.

Pfizer CT.gov Call Center

CONTACT

[email protected]

1-800-718-1021

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY

Important dates

Study start
2022
Primary completion
2040
Study completion
2040
First posted
Oct 6, 2022
Registry last updated
May 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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