Lifeline drug mitapivat keeps flowing for rare blood disorder patients
NCT ID NCT05777993
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does not test new treatments but ensures those who benefited can keep taking mitapivat.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
mitapivat (tablet)
What this could lead to
If successful, this study ensures uninterrupted treatment for people with pyruvate kinase deficiency who lack commercial access.
What could go wrong
This is a small rollover study (6 participants) focused on safety and continued access, not on proving new benefits. Side effects remain possible.
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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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
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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