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Tests treatment safety and results for Fabry Disease
Official title Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
ClinicalTrials.gov ID: NCT05710692
What this study is testing
What is PRX-102 1 mg/kg every 2 weeks?
PRX-102 1 mg/kg every 2 weeks is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Also referred to as pegunigalsidase alfa, Recombinant human alpha galactosidase-A.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 13 to 70
You may be able to join if
- (all people)
- 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...
- Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry...
You likely can't join if
- Administration of ERT for Fabry disease within 14 days before baseline, substrate reduction 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...
- 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...
- 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...
- Currently taking another investigational drug for any condition
See the full eligibility criteria
Who can join
- (all people)
- 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...
- 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 people: 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...
- 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 for people in Cohort A For people enrolled in Cohort A, these specific , in...
- 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...
- Aged ≥13 to \<18 years
- people who have previously received or are currently receiving ERT treatment, must be negative for ADAs to PRX-102
What rules you out
- 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...
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Chikushino-shi, Fukuoka, Japan
- Sendai, Miyagi, Japan
- Nishihara, Okinawa, Japan
- Suita, Osaka, Japan
- Bunkyo-ku, Tokyo, Japan
- Minato-ku, Tokyo, Japan
- Shinjuku-ku, Tokyo, Japan
- Asahikawa, Japan
- Niigata, Japan
- Okayama, Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 13 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chikushino-shi, Fukuoka, Japan; Sendai, Miyagi, Japan; Nishihara, Okinawa, Japan; Suita, Osaka, Japan; Bunkyo-ku, Tokyo, Japan; Minato-ku, Tokyo, Japan and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.