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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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.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Asahikawa Medical University Hospital

    RECRUITING

    Asahikawa, Japan

    Contact

    Contact

  • Fukuoka University Chikushi Hospital

    WITHDRAWN

    Chikushino-shi, Fukuoka, 818-8502, Japan

  • Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo

    RECRUITING

    Bunkyo-ku, Tokyo, 113-0033, Japan

    Contact

    Contact

  • Keio University Hospital

    RECRUITING

    Shinjuku-ku, Tokyo, 160-8582, Japan

    Contact

    Contact

  • National Hospital Organization Okayama Medical Center

    NOT_YET_RECRUITING

    Okayama, Japan

    Contact

    Contact

  • Niigata University Medical & Dental Hospital

    RECRUITING

    Niigata, 951-8520, Japan

    Contact

    Contact

  • Osaka University Hospital

    RECRUITING

    Suita, Osaka, 565-0871, Japan

    Contact

    Contact

  • Tohoku University Hospital

    RECRUITING

    Sendai, Miyagi, 980-8574, Japan

    Contact

    Contact

  • Tokyo Jikei University Hospital

    RECRUITING

    Minato-ku, Tokyo, 105-8461, Japan

    Contact

    Contact

  • University of the Ryukyu Hospital

    RECRUITING

    Nishihara, Okinawa, 903-0125, Japan

    Contact

    Contact