Lifeline drug access extended for rare blood disorder patients
NCT ID NCT05777993
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 20 times
Summary
This 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 is to monitor safety and side effects while providing continued treatment.
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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.
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
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Locations
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Kyoto Katsura Hospital
Kyoto, 615-8256, Japan
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McMaster Centre for Transfusion Medicine McMaster University
Hamilton, Ontario, L8S3Z5, Canada
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Toho University - Omori Medical Center
Ōta-ku, 143-8541, Japan
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Tohoku University Hospital
Sendai, Miyagi, 980-8574, Japan
Conditions
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