Lifeline drug access for rare blood disorder patients
NCT ID NCT05777993
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 23 times
Summary
This 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 monitor safety and side effects while providing ongoing treatment.
Disclaimer
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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.
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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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
Explore the condition pages connected to this study.