PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Gene therapy offers hope for rare blood disorder patients
Disease control TerminatedThis study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. About 10 participants will receive their own blood stem cells modified with a healthy gene to help produce more red blood cells. The goal is to raise hemoglobi…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 17, 2026 10:28 UTC
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Lifeline drug access extended for rare blood disorder patients
Disease control ENROLLING_BY_INVITATIONThis study offers ongoing access to the drug mitapivat for 6 adults with pyruvate kinase deficiency, a rare genetic blood condition causing anemia. Participants must have completed a prior Agios-sponsored mitapivat study and be unable to get the drug commercially. The main goal i…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE4 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 15, 2026 12:07 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 with pyruvate kinase deficiency, a rare red blood cell disorder. It will collect health data from about 500 participants worldwide over up to 9 years. The goal is to better understand how the disease progresses, how it is treated, and…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Agios Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 17, 2026 10:27 UTC
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Blood test study seeks hidden enzyme deficiency in cancer patients
Knowledge-focused OngoingThis study looks at how often people with certain blood cancers or related conditions have an acquired form of pyruvate kinase deficiency, a red blood cell enzyme problem. Researchers will take a single blood sample from 100 participants to check enzyme activity and gene changes.…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 15, 2026 11:57 UTC