Could a cancer drug make stem cell transplants safer for sickle cell patients?

NCT ID NCT07252050

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

Summary

This study tests whether adding the drug ruxolitinib to a standard stem cell transplant can prevent graft failure in children and young adults with severe sickle cell disease. Up to 24 participants will receive the drug during conditioning and be followed for two years after transplant. The goal is to improve survival without complications like graft failure or needing a second transplant.

What this could mean

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

Active substance
Ruxolitinib (Jakafi)
What this could lead to
If it works, this could make stem cell transplants safer and more effective for people with sickle cell disease, reducing the chance of graft failure.
What could go wrong
This is an early-phase trial with only 24 participants, so results may not apply to everyone. Adding ruxolitinib could also increase side effects like infection or bleeding.

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.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Colorado

    NOT_YET_RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Manning Family Children's

    NOT_YET_RECRUITING

    New Orleans, Louisiana, 70118, United States

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