AzTrials
AzTrials

Fabry DiseasePhase 2/3RISENCT05710692

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

A Phase 2/3 study testing PRX-102 1 mg/kg every 2 weeks and PRX-102 2 mg/kg every 4 weeks in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 10 sites in Japan.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 13 to 70 years
Conditions the study is for
Fabry Disease
What is being tested?
PRX-102 1 mg/kg every 2 weeks and PRX-102 2 mg/kg every 4 weeks
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
10 sitesJapan
Study dates
1 Aug 2023to Aug 2029
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 13 to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent
  • 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
  • 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.
Show all 32Show fewer
  • Clinical condition that in the opinion of the Investigator requires treatment with ERT
  • 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

Who cannot join

  • 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
Show all 15Show fewer
  • 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

Trial locations and contacts

10 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Chiesi Clinical Trial contact

+3905212791

clinicaltrials_info@chiesi.com

10 sites in 1 country

Japan10 sites, 9 recruiting

  • Asahikawa Medical University Hospital

    Asahikawa

    Naoki Nakagawa

    Naoki Nakagawa

    Recruiting

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

    Bunkyo-ku, Tokyo, 113-0033

    Takao Kato

    Takao Kato

    Recruiting

  • Fukuoka University Chikushi Hospital

    Chikushino-shi, Fukuoka, 818-8502

    Withdrawn

  • Tokyo Jikei University Hospital

    Minato-ku, Tokyo, 105-8461

    Masahisa Kobayashi

    Masahisa Kobayashi

    Recruiting

  • Niigata University Medical & Dental Hospital

    Niigata, 951-8520

    Hirofumi Watanabe

    Hirofumi Watanabe

    Recruiting

  • University of the Ryukyu Hospital

    Nishihara, Okinawa, 903-0125

    Koichi Nakanishi

    Koichi Nakanishi

    Recruiting

  • National Hospital Organization Okayama Medical Center

    Okayama

    Mahoko Furujo

    Mahoko Furujo

    Not yet recruiting

  • Tohoku University Hospital

    Sendai, Miyagi, 980-8574

    Saori Yamamoto

    Saori Yamamoto

    Recruiting

  • Keio University Hospital

    Shinjuku-ku, Tokyo, 160-8582

    Hiroyuki Yamakawa

    Hiroyuki Yamakawa

    Recruiting

  • Osaka University Hospital

    Suita, Osaka, 565-0871

    Tomoko Namba

    Tomoko Namba

    Recruiting

Other trials in Japan

About this study

Official title: 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)

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.

Read the detailed description

Investigators are doing this study to find out if treatment with pegunigalsidase alfa will prevent or reduce the development of health problems caused by Fabry disease and thereby improve patients' health and quality of life.

pegunigalsidase alfa (PRX-102) is a drug made using genetic engineering techniques and manufactured using cultured tobacco cells. It is given by intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight.

The study consists of a main study that is divided into two stages, each of which will last one year, followed by an optional extension study. In stage II of main study and in the optional extension study, the participants may receive PRX-102 intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight or every 4 weeks at a dosage of 2 milligrams per kilogram (mg/kg) of body weight.

There are three groups (cohorts) in this study, with adults enrolled in either Cohort A or B and adolescents in Cohort C. Whether an adult is assigned to Cohort A or Cohort B depends on their kidney function and treatment history.

This study will start with a screening visit of up to 6 weeks. It will be followed up by infusion visits every 2 weeks or 4 weeks. For subjects not continuing in the extension stage, a follow-up call is to be made 30 days after the last study drug infusion.

Conditions
Fabry Disease
Being tested
PRX-102 1 mg/kg every 2 weeks, PRX-102 2 mg/kg every 4 weeks
Medical subject headings
Fabry Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PRX-102 1 mg/kg every 2 weeksDrug

    Also called pegunigalsidase alfa, Recombinant human alpha galactosidase-A

    PRX-102 1 mg/kg every 2 weeks

  • PRX-102 2 mg/kg every 4 weeksDrug

    Also called pegunigalsidase alfa, Recombinant human alpha galactosidase-A

    PRX-102 2 mg/kg every 4 weeks

Study groups in NCT05710692
Study groupRoleWhat they receive
PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeksExperimentalPRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks (available only in the optional extension part)
Receives: Drug: PRX-102 1 mg/kg every 2 weeks; Drug: PRX-102 2 mg/kg every 4 weeks

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment Emergent Adverse Events (TEAEs)

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  2. Incidence of Infusion Related Reactions (IRRs)

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  3. Incidence of Injection site reactions (ISRs)

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  4. Change of laboratory tests' results

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  5. Change in in body weight in kilograms

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  6. Change in height in centimeters

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  7. Change in Tanner stage

    Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  8. Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, PR Interval, QRS Duration, QT Interval, QTc Interval, and ST Segment

    Quantitative ECG parameters will be summarized by cohort and overall

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  9. Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  10. ADA status change from baseline

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  11. Incidence of premedication use at each visit and change of infusion premedications from baseline

    Measured: 12 Months, 24 Months and through study completion (an average of 4.5 years)

  12. Change from baseline of Maximum plasma concentration (Cmax), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  13. Change from baseline of Time to maximum plasma concentration (tmax), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  14. Change from baseline of Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  15. Change from baseline of Area under the curve from time 0 to 2 weeks (AUC0-2wk), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  16. Change from baseline of Area under the curve from time 0 to infinity (AUC0-∞), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  17. Change from baseline of Terminal half-life (t1/2), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  18. Change from baseline of Area under the curve over a dosing interval (AUCτ), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  19. Change from baseline of Observed drug concentration at the end of the dosing interval (Cτ), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  20. Change from baseline of Clearance (Cl), pharmacokinetic parameter

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

  21. Change from baseline of Volume of distribution (Vz), pharmacokinetic parameters

    Measured: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Feb 2023
  2. Study start1 Aug 2023
  3. Primary completionOct 2027 (estimated)
  4. Study completionAug 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05710692
Sponsor's protocol ID
CLI-06657AA2-01
Acronym
RISE
Status last verified
Mar 2026
First submitted
13 Dec 2022
Last updated
18 Mar 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing PRX-102 1 mg/kg every 2 weeks and PRX-102 2 mg/kg every 4 weeks in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 10 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 32 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 18 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Chiesi Clinical Trial (+3905212791, clinicaltrials_info@chiesi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment Emergent Adverse Events (TEAEs) (time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)). The study lists 21 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05710692.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in