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One-Time gene therapy could change life for severe hemophilia patients

NCT ID NCT03061201

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested a new gene therapy (SB-525) in 13 adults with severe hemophilia A, a bleeding disorder. The goal was to see if a single infusion could safely raise the body's own clotting factor levels and reduce bleeding episodes. Participants were monitored for up to 5 years to track safety and how long the effect lasts.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

13 people

The number who actually took part.

Started

Jun 2017

Finished

Jul 2024

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: * Male ≥18 years of age * Severe hemophilia A (past evidence of circulating FVIII activity of \< 1% normal) * Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days * ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months * Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion Exclusion Criteria: * Presence of neutralizing antibodies * Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood) * History of hypersensitivity response to FVIII * History of Hepatitis B or HIV-1/2 infection * History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative * Evidence of any bleeding disorder in addition to hemophilia A * Markers of hepatic inflammation or overt or occult cirrhosis * History of chronic renal disease or creatinine ≥ 1.5 mg/dL * Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein * Presence of \> grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease

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Conditions

The condition(s) this trial relates to.

hemophilia A severe hemophilia A

As listed by the trial registrant

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

Hemophilia A

Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • City of Hope Medical Center

    Duarte, California, 91010, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Hemophilia Center of Western PA

    Pittsburgh, Pennsylvania, 15213, United States

  • Midtown Ambulatory Care Center

    Sacramento, California, 95817, United States

  • UC Davis Ambulatory Care Clinic

    Sacramento, California, 95817, United States

  • UC Davis CTSC Clinical Research Center

    Sacramento, California, 95817, United States

  • UC Davis Comprehensive Cancer Center

    Sacramento, California, 95817, United States

  • UC Davis Hemophilia Treatment Center

    Sacramento, California, 95817, United States

  • UC Davis Investigational Drug Services Pharmacy

    Sacramento, California, 95817, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UPMC Montefiore Clinical and Translational Research Center

    Pittsburgh, Pennsylvania, 15213, United States

  • UPMC, Investigational Drug Service

    Pittsburgh, Pennsylvania, 15213, United States

  • USF Health Morsani Center For Advanced Healthcare

    Tampa, Florida, 33612, United States

  • University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center

    Miami, Florida, 33136, United States

  • University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy

    San Francisco, California, 94143-0622, United States

  • University of California, San Francisco - Outpatient Hematology Clinic

    San Francisco, California, 94143, United States

  • University of California, San Francisco -Moffitt Hospital

    San Francisco, California, 94143, United States

  • Vanderbilt Hemostasis Treatment Clinic

    Nashville, Tennessee, 37232, United States

  • Vanderbilt Hemostasis-Thrombosis Clinic

    Nashville, Tennessee, 37232, United States

  • Vanderbilt University Medical Center Clinical Research Center

    Nashville, Tennessee, 37212-8646, United States

  • Washington Institute for Coagulation

    Seattle, Washington, 98101, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.