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Gene therapy for hemophilia: how safe is it years later?

NCT ID NCT05568719

Knowledge-focused Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 173 people with hemophilia A or B who previously received a single-dose gene therapy in a Pfizer trial. Researchers will track bleeding episodes, clotting factor levels, and any long-term side effects like blood clots or liver issues. The goal is to understand how well the treatment works over time under routine medical care.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gene therapy (giroctocogene fitelparvovec for hemophilia A, fidanacogene elaparvovec for hemophilia B)
What this could lead to
If successful, this study will confirm whether a single dose of gene therapy can safely control bleeding for years in people with hemophilia.
What could go wrong
This is a follow-up study, not a new treatment test. It only includes people who already received the gene therapy, so results may not apply to everyone. Long-term risks like liver problems or blood clots are being watched.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 173 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2022

Expected to finish

Feb 2040

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B Hemorrhage

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia A Hemophilia B

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    22 sites in 7 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Acibadem Adana Hospital

    NOT_YET_RECRUITING

    Adana, 01130, Turkey (Türkiye)

  • Cornell University||Joan & Sanford I. Weill Medical College

    RECRUITING

    New York, New York, 10065, United States

  • Ege Universitesi Hastanesi

    RECRUITING

    Izmir, İ̇zmir, 35100, Turkey (Türkiye)

  • Kyung Hee University Hospital at Gangdong

    NOT_YET_RECRUITING

    Seoul, 05278, South Korea

  • Mississippi Center For Advanced Medicine

    TERMINATED

    Madison, Mississippi, 39110, United States

  • National Taiwan University Hospital

    NOT_YET_RECRUITING

    Taipei, 10041, Taiwan

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Skåne University Hospital

    NOT_YET_RECRUITING

    Malmö, 21428, Sweden

  • Sydney Local Health District (RPAH Zone)

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Taichung Veterans General Hospital

    NOT_YET_RECRUITING

    Taichung, 40705, Taiwan

  • The Childrens Hospital of Philadelphia Division of Hematology

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • The Childrens Hospital of Philadelphia Division of Hematology

    RECRUITING

    Philadelphia, Pennsylvania, 19146, United States

  • The Regents of the University of California||UC Davis Health

    RECRUITING

    Rancho Cordova, California, 95670, United States

  • The University of South Florida Board of Trustees

    RECRUITING

    Tampa, Florida, 33620, United States

  • UC Davis Ambulatory Care Clinic

    RECRUITING

    Sacramento, California, 95817, United States

  • UC Davis Health

    RECRUITING

    Sacramento, California, 95816, United States

  • UC Davis Hemophilia Treatment Center

    RECRUITING

    Sacramento, California, 95817, United States

  • UC Davis Medical Center

    RECRUITING

    Sacramento, California, 95817, United States

  • UCSF Outpatient Hematology Clinic

    NOT_YET_RECRUITING

    San Francisco, California, 94143, United States

  • USF Health Morsani Center For Advanced Healthcare

    RECRUITING

    Tampa, Florida, 33612, United States

  • Unity Health Toronto, St. Michael's Hospital

    NOT_YET_RECRUITING

    Toronto, Ontario, M5B 1W8, Canada

  • Washington Institute for Coagulation

    NOT_YET_RECRUITING

    Seattle, Washington, 98101, United States

  • Weill Cornell Medical College-New York Presbyterian Hospital

    RECRUITING

    New York, New York, 10065, United States

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