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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
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
Secondary ID [1] 0 0
C0371017
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Diagnosis / Prognosis
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of thromboembolic events
Timepoint [1] 0 0
Day 1 to 10 years
Primary outcome [2] 0 0
Incidence of factor inhibitor development
Timepoint [2] 0 0
Day 1 to 10 years
Primary outcome [3] 0 0
Incidence of hepatic malignancy
Timepoint [3] 0 0
Day 1 to 10 years
Primary outcome [4] 0 0
Incidence of liver abnormalities
Timepoint [4] 0 0
Day 1 to 10 years
Primary outcome [5] 0 0
Factor activity level
Timepoint [5] 0 0
Day 1 to 10 years
Secondary outcome [1] 0 0
Total ABR (treated or untreated; (excluding bleeds related to surgery)
Timepoint [1] 0 0
Day 1 to 10 years
Secondary outcome [2] 0 0
Incidence of and time from vector infusion to resumption of prophylaxis
Timepoint [2] 0 0
Day 1 to 10 years
Secondary outcome [3] 0 0
AIR of exogenous factor (excluding infusions related to surgery)
Timepoint [3] 0 0
Day 1 to 10 years
Secondary outcome [4] 0 0
Consumption of exogenous factor (excluding infusions related to surgery)
Timepoint [4] 0 0
Day 1 to 10 years
Secondary outcome [5] 0 0
Incidence of Non-hepatic malignancy
Timepoint [5] 0 0
Day 1 to 10 years
Secondary outcome [6] 0 0
Incidence of Auto-immune disorders
Timepoint [6] 0 0
Day 1 to 10 years
Secondary outcome [7] 0 0
Incidence of SAEs
Timepoint [7] 0 0
Day 1 to 10 years
Secondary outcome [8] 0 0
All cause mortality
Timepoint [8] 0 0
Day 1 to 10 years
Secondary outcome [9] 0 0
EQ-5D-5L dimension and VAS scores
Timepoint [9] 0 0
Day 1 to 10 years

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.
Minimum age
18 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
-None

Study design
Purpose of the study
Other
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
3/09/2039
Actual
Sample size
Target
263
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Mississippi
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Washington
Country [6] 0 0
Turkey
State/province [6] 0 0
I?zmir

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Country 0 0
Phone 0 0
1-800-718-1021
Fax 0 0
Email 0 0
ClinicalTrials.gov_Inquiries@pfizer.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


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