Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05568719




Registration number
NCT05568719
Ethics application status
Date submitted
3/10/2022
Date registered
6/10/2022
Date last updated
17/05/2024

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
8/04/2038
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
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Mississippi
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
United States of America
State/province [4] 0 0
Washington
Country [5] 0 0
Turkey
State/province [5] 0 0
I?zmir

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

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05568719
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.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05568719