A Non-Investigational Product, Multi-Center Cohort Study to Describe Long-Term Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pfizer Inc.

Participant type

Patients

Age range

18-64 years

Gender

Male

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2026-04-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Pfizer Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Efficacy

To characterize the long-term safety of participants who have received prior treatment with giroctocogene fitelparvovec or fidanacogene elaparvovec
To describe durability of transgene expression of giroctocogene fitelparvovec or fidanacogene elaparvovec

Secondary objectives 3

1. To assess the effect of giroctocogene fitelparvovec or fidanacogene elaparvovec on clinical outcomes
2. To further evaluate the long-term safety profile of giroctocogene fitelparvovec or fidanacogene elaparvovec in participants
3. To evaluate the effect of giroctocogene fitelparvovec or fidanacogene elaparvovec on the participant’s quality of life and function

Conditions and MedDRA coding

hemophilia A

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-002724-PIP01-19, EMEA-002362-PIP02-19

Plan to share IPD

Yes

IPD 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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Only participants who received giroctocogene fitelparvovec or fidanacogene elaparvovec and were enrolled in a Pfizer-sponsored study are eligible.

Exclusion criteria 1

1. None

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Incidence of thromboembolic events
2. Incidence of factor inhibitor development
3. Incidence of hepatic malignancy
4. Incidence of liver abnormalities
5. Factor activity level

Secondary endpoints 9

1. Total ABR (treated and untreated)
2. Incidence of and time from previously administered gene therapy infusion to resumption of prophylaxis
3. AIR of exogenous factor
4. Consumption of exogenous factor
5. Incidence of Non-hepatic malignancy
6. Incidence of Auto-immune disorders
7. Incidence of SAEs
8. All cause-mortality
9. EQ-5D-5L dimension and VAS scores

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation

Pfizer Inc.

Address

66 Hudson Boulevard East

City

New York

Postcode

10001-2189

Country

United States

Scientific contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Third parties 9

Locations

6 EU/EEA countries · 9 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 35 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications