New hope for fabry disease: enzyme therapy trial launches in japan
NCT ID NCT05710692
First seen Dec 08, 2025 · Last updated May 13, 2026 · Updated 20 times
Summary
This study tests a new enzyme replacement therapy called pegunigalsidase alfa in Japanese patients with Fabry disease, a rare genetic disorder. About 16 people aged 13 to 70 will receive the treatment to see if it is safe and helps control the disease. The goal is to manage symptoms and prevent complications, not to cure the condition.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Asahikawa Medical University Hospital
RECRUITINGAsahikawa, Japan
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Fukuoka University Chikushi Hospital
WITHDRAWNChikushino-shi, Fukuoka, 818-8502, Japan
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Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
RECRUITINGBunkyo-ku, Tokyo, 113-0033, Japan
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Keio University Hospital
RECRUITINGShinjuku-ku, Tokyo, 160-8582, Japan
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National Hospital Organization Okayama Medical Center
NOT_YET_RECRUITINGOkayama, Japan
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Niigata University Medical & Dental Hospital
RECRUITINGNiigata, 951-8520, Japan
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Osaka University Hospital
RECRUITINGSuita, Osaka, 565-0871, Japan
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Contact
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Tohoku University Hospital
RECRUITINGSendai, Miyagi, 980-8574, Japan
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Contact
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Tokyo Jikei University Hospital
RECRUITINGMinato-ku, Tokyo, 105-8461, Japan
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Contact
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University of the Ryukyu Hospital
RECRUITINGNishihara, Okinawa, 903-0125, Japan
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Conditions
Explore the condition pages connected to this study.