New hope for fabry disease: japanese trial launches for enzyme therapy
NCT ID NCT05710692
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing a drug called pegunigalsidase alfa in about 16 Japanese patients aged 13 to 70 with Fabry disease, a rare genetic disorder. The goal is to see if the drug is safe and how it works in the body. Participants will receive the treatment and be monitored for side effects and changes in lab results.
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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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Asahikawa Medical University Hospital
RECRUITINGAsahikawa, Japan
Contact
Contact
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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
Contact
Contact
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Keio University Hospital
RECRUITINGShinjuku-ku, Tokyo, 160-8582, Japan
Contact
Contact
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National Hospital Organization Okayama Medical Center
NOT_YET_RECRUITINGOkayama, Japan
Contact
Contact
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Niigata University Medical & Dental Hospital
RECRUITINGNiigata, 951-8520, Japan
Contact
Contact
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Osaka University Hospital
RECRUITINGSuita, Osaka, 565-0871, Japan
Contact
Contact
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Tohoku University Hospital
RECRUITINGSendai, Miyagi, 980-8574, Japan
Contact
Contact
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Tokyo Jikei University Hospital
RECRUITINGMinato-ku, Tokyo, 105-8461, Japan
Contact
Contact
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University of the Ryukyu Hospital
RECRUITINGNishihara, Okinawa, 903-0125, Japan
Contact
Contact