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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05568719
Registration number
NCT05568719
Ethics application status
Date submitted
3/10/2022
Date registered
6/10/2022
Titles & IDs
Public title
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
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Scientific 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
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Secondary ID [1]
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C0371017
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
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Hemophilia B
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Diagnosis / Prognosis - Testing of hepatic AAV Vector integration
Other: 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
Other: 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
Diagnosis / Prognosis: 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
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Intervention code [1]
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Diagnosis / Prognosis
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of thromboembolic events
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Assessment method [1]
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Timepoint [1]
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Day 1 to 10 years
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Primary outcome [2]
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Incidence of factor inhibitor development
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Assessment method [2]
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FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).
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Timepoint [2]
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Day 1 to 10 years
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Primary outcome [3]
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Incidence of hepatic malignancy
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Assessment method [3]
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0
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Timepoint [3]
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Day 1 to 10 years
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Primary outcome [4]
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Incidence of liver abnormalities
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Assessment method [4]
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Timepoint [4]
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Day 1 to 10 years
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Primary outcome [5]
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Factor activity level
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Assessment method [5]
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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.
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Timepoint [5]
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Day 1 to 10 years
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Secondary outcome [1]
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Total ABR (treated or untreated; (excluding bleeds related to surgery)
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Assessment method [1]
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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.
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Timepoint [1]
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Day 1 to 10 years
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Secondary outcome [2]
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Incidence of and time from vector infusion to resumption of prophylaxis
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Assessment method [2]
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Describe incidence of resumption of prophylaxis resumption and the time (in days) to resumption of prophylaxis after receiving vector infusion.
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Timepoint [2]
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Day 1 to 10 years
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Secondary outcome [3]
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AIR of exogenous factor (excluding infusions related to surgery)
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Assessment method [3]
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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.
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Timepoint [3]
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Day 1 to 10 years
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Secondary outcome [4]
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Consumption of exogenous factor (excluding infusions related to surgery)
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Assessment method [4]
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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
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Timepoint [4]
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Day 1 to 10 years
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Secondary outcome [5]
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Incidence of Non-hepatic malignancy
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Assessment method [5]
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Timepoint [5]
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Day 1 to 10 years
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Secondary outcome [6]
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Incidence of Auto-immune disorders
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Assessment method [6]
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0
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Timepoint [6]
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Day 1 to 10 years
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Secondary outcome [7]
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Incidence of SAEs
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Assessment method [7]
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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.
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Timepoint [7]
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Day 1 to 10 years
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Secondary outcome [8]
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All cause mortality
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Assessment method [8]
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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.
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Timepoint [8]
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Day 1 to 10 years
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Secondary outcome [9]
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EQ-5D-5L dimension and VAS scores
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Assessment method [9]
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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
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Timepoint [9]
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Day 1 to 10 years
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Eligibility
Key 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.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
-None
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Study design
Purpose of the study
Other
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/12/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
3/09/2039
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Actual
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Sample size
Target
263
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Camperdown
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Florida
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Country [2]
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United States of America
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State/province [2]
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Mississippi
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Country [3]
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United States of America
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State/province [3]
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Pennsylvania
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Country [4]
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United States of America
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State/province [4]
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Washington
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Country [5]
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Turkey
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State/province [5]
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I?zmir
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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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Trial website
https://clinicaltrials.gov/study/NCT05568719
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Trial related presentations / publications
Berntorp E, Shapiro AD. Modern haemophilia care. Lancet. 2012 Apr 14;379(9824):1447-56. doi: 10.1016/S0140-6736(11)61139-2. Epub 2012 Mar 27. WFH guidelines: https://www1.wfh.org/publication/files/pdf-1472.pdf Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. No abstract available. Blanchette VS, McCready M, Achonu C, Abdolell M, Rivard G, Manco-Johnson MJ. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia. 2003 May;9 Suppl 1:19-26; discussion 26. doi: 10.1046/j.1365-2516.9.s1.12.x. Lillicrap D. Extending half-life in coagulation factors: where do we stand? Thromb Res. 2008;122 Suppl 4:S2-8. doi: 10.1016/S0049-3848(08)70027-6.
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Public notes
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Contacts
Principal investigator
Name
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Pfizer CT.gov Call Center
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Address
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Pfizer
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Pfizer CT.gov Call Center
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Address
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Country
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Phone
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1-800-718-1021
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05568719