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A Study to Learn About the Safety and Effectiveness of the Study Medicines Called Giroctocogene Fitelparvovec and Fidanacogene Elaparvovec

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05568719
Recruitment Status : Recruiting
First Posted : October 6, 2022
Last Update Posted : December 5, 2022
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
A low-intervention 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.

Condition or disease Intervention/treatment
Hemophilia A Hemophilia B Diagnostic Test: Testing of hepatic AAV Vector integration

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY, LOW- INTERVENTIONAL 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
Estimated Study Start Date : January 13, 2023
Estimated Primary Completion Date : October 19, 2037
Estimated Study Completion Date : October 19, 2037

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
Hemophilia A / giroctocogene fitelparvovec
Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study
Diagnostic Test: Testing of hepatic AAV Vector integration
Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated

Hemophilia B / fidanacogene elaparvovec
Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study
Diagnostic Test: Testing of hepatic AAV Vector integration
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 Outcome Measures :
  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 Outcome Measures :
  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:

    1. Mobility
    2. Self-care
    3. Usual activities
    4. Pain/Discomfort
    5. Anxiety/Depression



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male participants over the age of 18 with Hemophilia A or Hemophilia B who have received prior treatment with investigational giroctocogene fitelparvovec or fidanacogene eleparvovec, respectively.
Criteria

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05568719


Contacts
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Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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United States, Mississippi
Mississippi Center For Advanced Medicine Recruiting
Madison, Mississippi, United States, 39110
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
Publications:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT05568719    
Other Study ID Numbers: C0371017
First Posted: October 6, 2022    Key Record Dates
Last Update Posted: December 5, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pfizer:
bleeding
adeno-associated virus based vector
gene therapy
giroctocogene fitelparvovec
fidanacogene elaparvovec
factor VIII
factor IX
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked