PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Lifeline drug access for rare blood disorder patients
Disease control ENROLLING_BY_INVITATIONThis study offers continued access to the drug mitapivat for 6 adults with pyruvate kinase deficiency, a rare genetic blood condition that causes anemia. Participants must have completed a prior mitapivat study and be unable to get the drug commercially. The main goal is to monit…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE4 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 26, 2026 10:44 UTC
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Gene therapy could free patients from frequent blood transfusions
Disease control TerminatedThis trial tests a one-time gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Patients receive their own blood stem cells modified to produce a working enzyme. The goal is to raise hemoglobin levels and reduce or eliminate the n…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 26, 2026 10:31 UTC
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Global registry launches to unlock secrets of rare blood disorder
Knowledge-focused OngoingThis study is a long-term registry for people of all ages with pyruvate kinase deficiency, a rare red blood cell disorder. It does not test any new treatments. Instead, researchers will collect health information from doctors and patients over up to 9 years to better understand h…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Agios Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 22, 2026 14:02 UTC
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Blood test study seeks hidden enzyme defect in cancer patients
Knowledge-focused OngoingThis study looks at how common acquired pyruvate kinase deficiency is in people with certain blood cancers or pre-cancer conditions. Researchers will take a single blood sample from 100 participants to check for low enzyme activity or gene changes. The goal is to better understan…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 18, 2026 12:03 UTC