Gene therapy offers hope for rare blood disorder
NCT ID NCT06422351
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Ten participants will receive their own blood stem cells modified with a healthy gene to help produce normal red blood cells. The goal is to raise hemoglobin levels and reduce the need for blood transfusions.
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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
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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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Hospital Universitario Fundación Jiménez Díaz
Madrid, Spain
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Stanford University
Palo Alto, California, 94305, United States
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Other studies related to the condition(s) this trial covers.
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