New hope for fabry disease: japanese trial launches for enzyme therapy

NCT ID NCT05710692

Recruiting now Disease control Sponsor: Chiesi Farmaceutici S.p.A. Source: ClinicalTrials.gov ↗

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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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.

FABRY DISEASE

Contacts and locations

Locations

Asahikawa Medical University Hospital
RECRUITING

Asahikawa, Japan
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WITHDRAWN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
Keio University Hospital
RECRUITING

Shinjuku-ku, Tokyo, 160-8582, Japan
National Hospital Organization Okayama Medical Center
NOT_YET_RECRUITING

Okayama, Japan
Niigata University Medical & Dental Hospital
RECRUITING

Niigata, 951-8520, Japan
Osaka University Hospital
RECRUITING

Suita, Osaka, 565-0871, Japan
Tohoku University Hospital
RECRUITING

Sendai, Miyagi, 980-8574, Japan
Tokyo Jikei University Hospital
RECRUITING

Minato-ku, Tokyo, 105-8461, Japan
University of the Ryukyu Hospital
RECRUITING

Nishihara, Okinawa, 903-0125, Japan

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