Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

Recruiting now · Phase 2/Phase 3

Conditions studied: Fabry Disease

In brief

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.

Key facts

Study ID

NCT05710692

Run by

Chiesi Farmaceutici S.p.A.

People needed

16

Starts

2023-08-01

Expected to finish

2029-08-01

Last updated by the study team

2026-03-18

Who can join

Age: 13 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Administration of ERT for Fabry disease within 14 days before baseline, substrate reduction therapy for Fabry disease within 3 days before baseline, or chaperone therapy for Fabry disease within 3 days before baseline
• History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug
• Cohort A only: eGFR value of >90 to ≤120 mL/min/1.73 m2 at screening and a historical eGFR value >120 mL/min/1.73 m2 in the 9 to 24 months before screening, indicating absence of renal impairment. eGFR to be calculated using the JPN-CKD-EPI creatinine equation (2009).
• Urine protein to creatinine ratio (UPCR) >0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB
• Initiation of treatment, or a change in dose to ongoing treatment, with an angiotensin-converting-enzyme inhibitor (ACEI) or angiotensin II receptor blocker (ARB) in the 4 weeks prior to screening.
• Currently taking another investigational drug for any condition
• Carry only known non-pathogenic Fabry mutations
• History of renal dialysis or kidney transplantation
• History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and renal vasculitis); non-specific conditions (e.g., ischemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, and acute postrenal obstructive nephropathy
• History of (or current) malignancy requiring treatment; the one exception is a prior history of resected basal cell carcinoma
• Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening
• A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within 3 months prior to screening, using a validated molecular assay or validated antigen assay
• Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion received
• Presence of any medical, emotional, behaevioral, or psychological condition that in the judgment of the Investigator could interfere with the subject's compliance with the requirements of the study
• Previous treatment with cellular therapy or gene therapy for any condition

Where it is running

• Niigata University Medical & Dental Hospital — Niigata, Japan (enrolling)
• Tohoku University Hospital — Sendai, Miyagi, Japan (enrolling)
• University of the Ryukyu Hospital — Nishihara, Okinawa, Japan (enrolling)
• Osaka University Hospital — Suita, Osaka, Japan (enrolling)
• Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo — Bunkyo-ku, Tokyo, Japan (enrolling)
• Tokyo Jikei University Hospital — Minato-ku, Tokyo, Japan (enrolling)
• Keio University Hospital — Shinjuku-ku, Tokyo, Japan (enrolling)
• Asahikawa Medical University Hospital — Asahikawa, Japan (enrolling)
• National Hospital Organization Okayama Medical Center — Okayama, Japan
• Fukuoka University Chikushi Hospital — Chikushino-shi, Fukuoka, Japan

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.