jRCT2031230079招募中不适用
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)
Chiesi Farmaceutici S.p.A. (ICCC: ICON Clinical Research GK)0 个研究点目标入组 18 人开始时间: 2023年8月25日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 13age old over 至 60age old under（—）
- 性别
- All
入选标准
- •Subjects must meet all of the following inclusion criteria to be eligible for enrolment in the study:
- •All subjects
- •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 alfa-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 m^
- •For adults, this will be calculated using the Chronic Kidney Disease Epidemiology Collaboration (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.
- •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:
- •a. Have a negative serum pregnancy test result at screening, AND
- •b. Agree to undergo a urine pregnancy test at baseline and every 12 weeks thereafter up to the final treatment, AND
- •c. 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 releasing system (IUS)
- •Combined (both estrogen 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
- •Aged =>18 to =< 60 years
- •Treatment with agalsidase beta 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 or equal to -2 mL/min/1.73 m~2/year. The historical slope will be calculated based on at least 3 serum creatinine values obtained over the past 9 to 24 months prior to screening, using the CKD-EPI Creatinine equation (2009). It is preferable if all 3 samples assessed were done at the same center using the same laboratory; however, the results of samples that were assessed by different laboratories will be accepted. 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
- •Aged =>18 to =< 60 years
- •Additional inclusion criteria for subjects in Cohort C
- •Aged =>13 to <18 years
- •If they previously received or are currently receiving ERT treatment, the subjects must be negative for anti-drug antibodies for PRX-102
- •Before the start of treatment, the Investigator will decide whether or not pregnancy testing and contraception counselling are necessary. Since over the course of the study, pre-pubertal girls may reach menarche and adolescents of either gender may become sexually active, the Investigator must periodically check on the status of these issues and implement pregnancy testing and/or contraception counselling if required.
排除标准
- •The presence of any of the following will exclude a prospective subject from study enrolment:
- •Administration of previous treatment 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 severe 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 m^2 at screening, and a historical eGFR value >120 mL/min/1.73 m^2 in the past 9 to 24 months before screening, indicating absence of renal impairment
- •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); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy
- •History of (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 study participation ends
- •Presence of any medical, emotional, behavioral, or psychological condition that in the judgment of the Investigator could interfere with the subject compliance with the requirements of the study
- •Previous treatment with cellular therapy or gene therapy for any condition
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