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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

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

A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (NCT05710692) is a Phase 2 / Phase 3 interventional studying Fabry Disease, sponsored by Chiesi Farmaceutici S.p.A.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Fabry Disease and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 16 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Inclusion criteria (all subjects) 1. Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent 2. A documented diagnosis of Fabry disease, as determined by the following: - Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene - Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease - All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma 3. Estimated glomerular filtration rate (eGFR) at screening ≥40 mL/min/1.73 m2. For adults, this will be calculated using the Japanese Modified Chronic Kidney Disease Epidemiology Collaboration (JPN-CKD-EPI) Creatinine equation (2009); and for adolescents, it will be calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation. 4. Clinical condition that in the opinion of the Investigator requires treatment with ERT 5. A female subject (including an adolescent in Cohort C, if applicable) must meet one of the following criteria: - If of childbearing potential, she must: - Have a negative serum pregnancy test result at screening, AND - Agree to undergo a urine pregnancy test at baseline and every 12 weeks thereafter up to the final treatment, AND - Agree to use one of the following highly reliable methods of contraception from the day of the willing to sign a consent form signature until 30 days after the last infusion received. The following methods are acceptable: - Placement of an intrauterine device (IUD) or intrauterine system (IUS) ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
Inclusion criteria (all subjects) 1. Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent 2. A documented diagnosis of Fabry disease, as determined by the following: * Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene * Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease * All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma 3. Estimated glomerular filtration rate (eGFR) at screening ≥40 mL/min/1.73 m2. For adults, this will be calculated using the Japanese Modified Chronic Kidney Disease Epidemiology Collaboration (JPN-CKD-EPI) Creatinine equation (2009); and for adolescents, it will be calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation. 4. Clinical condition that in the opinion of the Investigator requires treatment with ERT 5. A female subject (including an adolescent in Cohort C, if applicable) must meet one of the following criteria: * If of childbearing potential, she must: * Have a negative serum pregnancy test result at screening, AND * Agree to undergo a urine pregnancy test at baseline and every 12 weeks thereafter up to the final treatment, AND * Agree to use one of the following highly reliable methods of contraception from the day of the informed consent signature until 30 days after the last infusion received. The following methods are acceptable: * Placement of an intrauterine device (IUD) or intrauterine system (IUS) * Combined (both oestrogen and progestogen) hormonal contraception (oral) associated with inhibition of ovulation, supplemented with a barrier method (preferably male condom) * Bilateral tubal occlusion * Sexual abstinence, defined as refraining from heterosexual intercourse during the entire study period * Partner vasectomy, provided that the partner is the sole sexual partner and has received medical verification of the surgical success * Be of non-childbearing potential, defined as one of the following: * Post-menopausal (12 consecutive months of amenorrhea), OR * Permanently sterile following hysterectomy, bilateral salpingectomy, or bilateral oophorectomy (supporting evidence required) Additional inclusion criteria for subjects in Cohort A For subjects enrolled in Cohort A, these specific inclusion criteria, in addition to those above, apply: * Aged ≥18 to ≤70 years * Treatment with agalsidase beta or agalsidase alfa for at least the last 12 months prior to screening, with the dose stable (defined as having received at least 80% of the labelled dose) for at least the last 6 months * Diagnosis of kidney impairment, defined as a linear slope of eGFR more negative than or equal to -2 mL/min/1.73 m2/year. The historical eGFR slope will be calculated based on at least 3 serum creatinine values obtained over the 9 to 24 months prior to screening, using the JPN-CKD-EPI Creatinine equation (2009). This criterion will be confirmed at screening by calculating the screening eGFR slope using historical and screening serum creatinine values. Both historical and screening eGFR slopes will be used for the diagnosis of kidney impairment. Additional inclusion criterion for subjects in Cohort B For subjects enrolled in Cohort B, this specific inclusion criterion, in addition to those above, applies: \- Aged ≥18 to ≤70 years Additional inclusion criteria for subjects in Cohort C For subjects enrolled in Cohort C, these specific inclusion criteria, in addition to those above, apply: * Aged ≥13 to \<18 years * Subjects who have previously received or are currently receiving ERT treatment, must be negative for ADAs to PRX-102 Exclusion Criteria: 1. 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 2. 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 3. 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). 4. 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 5. 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. 6. Currently taking another investigational drug for any condition 7. Carry only known non-pathogenic Fabry mutations 8. History of renal dialysis or kidney transplantation 9. 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 10. History of (or current) malignancy requiring treatment; the one exception is a prior history of resected basal cell carcinoma 11. Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening 12. 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 13. 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 14. 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 15. Previous treatment with cellular therapy or gene therapy for any condition

Treatments Being Tested

DRUG

PRX-102 1 mg/kg every 2 weeks

PRX-102 1 mg/kg every 2 weeks

DRUG

PRX-102 2 mg/kg every 4 weeks

PRX-102 2 mg/kg every 4 weeks

Locations (10)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT05710692), the sponsor (Chiesi Farmaceutici S.p.A.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT05710692 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT05710692?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT05710692?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT05710692. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT05710692. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.