Actively Recruiting
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Led by Chiesi Farmaceutici S.p.A. · Updated on 2026-03-18
16
Participants Needed
10
Research Sites
313 weeks
Total Duration
On this page
Sponsors
C
Chiesi Farmaceutici S.p.A.
Lead Sponsor
I
ICON plc
Collaborating Sponsor
AI-Summary
What this Trial Is About
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.
CONDITIONS
Official Title
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Born in Japan with biological parents and all four grandparents of Japanese descent
- Diagnosed with Fabry disease confirmed by enzyme activity or genetic testing
- Presence of at least one Fabry disease feature: neuropathic pain, cornea verticillata, or clustered angiokeratoma
- Estimated glomerular filtration rate (eGFR) at screening of 40 mL/min/1.73 m2 or higher
- Clinical condition requiring treatment with enzyme replacement therapy (ERT)
- Female participants of childbearing potential must have a negative pregnancy test at screening, agree to regular pregnancy tests, and use reliable contraception until 30 days after the last infusion
- Female participants not of childbearing potential must be post-menopausal or permanently sterile
- Cohort A adults aged 18 to 70 years with stable treatment and kidney impairment
- Cohort B adults aged 18 to 70 years
- Cohort C adolescents aged 13 to under 18 years, negative for anti-drug antibodies to PRX-102 if previously or currently receiving ERT
You will not qualify if you...
- Received ERT within 14 days, substrate reduction therapy within 3 days, or chaperone therapy within 3 days before baseline
- History of severe allergic reactions to ERT or study drug components
- Cohort A adults with eGFR values indicating no kidney impairment
- Urine protein to creatinine ratio over 0.5 g/g without treatment with ACE inhibitors or ARBs
- Recent start or dose change of ACE inhibitors or ARBs within 4 weeks before screening
- Currently taking another investigational drug
- Carry only non-pathogenic Fabry mutations
- History of kidney dialysis or transplant
- Acute kidney injury within 12 months before screening
- History or current malignancy needing treatment, except treated basal cell carcinoma
- Severe cardiomyopathy or unstable heart disease within 6 months before screening
- Positive COVID-19 test within 3 months before screening
- Females pregnant, lactating, or unwilling to use contraception
- Any medical, emotional, behavioral, or psychological condition interfering with study compliance
- Previous treatment with cellular or gene therapy for any condition
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
Fukuoka University Chikushi Hospital
Chikushino-shi, Fukuoka, Japan, 818-8502
Withdrawn
2
Tohoku University Hospital
Sendai, Miyagi, Japan, 980-8574
Actively Recruiting
3
University of the Ryukyu Hospital
Nishihara, Okinawa, Japan, 903-0125
Actively Recruiting
4
Osaka University Hospital
Suita, Osaka, Japan, 565-0871
Actively Recruiting
5
Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
Bunkyo-ku, Tokyo, Japan, 113-0033
Actively Recruiting
6
Tokyo Jikei University Hospital
Minato-ku, Tokyo, Japan, 105-8461
Actively Recruiting
7
Keio University Hospital
Shinjuku-ku, Tokyo, Japan, 160-8582
Actively Recruiting
8
Asahikawa Medical University Hospital
Asahikawa, Japan
Actively Recruiting
9
Niigata University Medical & Dental Hospital
Niigata, Japan, 951-8520
Actively Recruiting
10
National Hospital Organization Okayama Medical Center
Okayama, Japan
Not Yet Recruiting
Research Team
C
Chiesi Clinical Trial
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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