Gene therapy trial hopes to ease hemophilia a

NCT ID NCT07226206

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

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Center for Inherited Blood Disorders

    RECRUITING

    Orange, California, 92868-4748, United States

  • Kaiser Permanente

    RECRUITING

    Vallejo, California, 94589-2441, United States