Gene therapy trial hopes to ease hemophilia a

NCT ID NCT07226206

Recruiting now Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 05, 2026 · Updated 4 times

Summary

This study tests a new gene therapy called SPK-8011QQ in 5 adult men with severe or moderately severe hemophilia A. The goal is to see if it is safe and tolerable. Participants receive a one-time intravenous dose, and researchers monitor for side effects and lab changes.

What this could mean

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

Active substance: SPK-8011QQ (gene therapy)
What this could lead to: If successful, this could point toward a one-time gene therapy that reduces or eliminates the need for regular factor VIII infusions for people with severe hemophilia A.
What could go wrong: This is a very early, small trial (only 5 participants) focused on safety, not effectiveness. Gene therapies can have unexpected side effects, and it may not work for everyone.

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

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Conditions

The condition(s) this trial relates to.

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

Center for Inherited Blood Disorders
RECRUITING

Orange, California, 92868-4748, United States
Kaiser Permanente
RECRUITING

Vallejo, California, 94589-2441, United States
University of California San Francisco
RECRUITING

San Francisco, California, 94143-1222, United States

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