New gene therapy aims to free patients from sickle cell pain crises

NCT ID NCT07432867

Summary

This study is testing a new gene therapy called DREAM01 for people with severe sickle cell disease who don't have a matched sibling donor for a bone marrow transplant. Doctors take a patient's own blood stem cells, genetically modify them in a lab to produce healthy hemoglobin, and then transplant them back. The goal is to reduce or eliminate the painful blockages in blood vessels (vaso-occlusive crises) and prevent long-term organ damage.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Department of Biotherapy, Necker-Enfants Malades Hospital

    RECRUITING

    Paris, Île-de-France Region, 75015, France

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

Conditions

Explore the condition pages connected to this study.