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One-Shot CRISPR cure trial for devastating blood diseases

NCT ID NCT05477563

Recruiting now ⭐️ CURE ⭐️ Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

Summary

This study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful crises in sickle cell patients. Researchers will monitor 26 participants to see if the treatment is safe and effective at correcting the underlying genetic cause of these diseases.

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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Contacts and locations

Study contacts

  • Contact

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

Locations

  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

    RECRUITING

    Rome, Italy

  • King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology

    RECRUITING

    Al Mathar Ash Shamali, Saudi Arabia

  • Levine Children's Hospital - Hematology

    RECRUITING

    Charlotte, North Carolina, 28203, United States

  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital

    RECRUITING

    New York, New York, 10032, United States

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    RECRUITING

    Nashville, Tennessee, 37203, United States

  • University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    RECRUITING

    Düsseldorf, Germany

Conditions

Explore the condition pages connected to this study.

BETA-THALASSEMIA GENETIC DISEASES, INBORN HEMATOLOGIC DISEASES HEMOGLOBINOPATHIES SICKLE CELL ANEMIA SICKLE CELL DISEASE THALASSEMIA