ISRCTN14012649进行中（未招募）3 期
Multi-centre, open-label trial to assess the saFety, pharmacodynamics, efficacy and pharmacokinetics of pegunigaLsidase alfa in patients from 2 years to less than 18 years of age with confirmed FabrY disease (FLY)
Chiesi (Italy)0 个研究点目标入组 22 人开始时间: 2024年7月29日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 22
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Male or female aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to <18 years (Cohort C)
- •2. A documented diagnosis of Fabry disease, as determined by the following:
- •2.1. Males: Plasma and/or leukocyte alpha-galactosidase-A (a-GAL-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 a-GAL-A (GLA)gene.
- •2.2. Females: Historical genetic test results consistent with Fabry mutations, or, in the case of novel mutations, a first-degree male relative with Fabry disease.
- •2.3. All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
- •3. History of Fabry pain:
- •3.1. Episodic crises (Fabry crises) characterised by agonizing burning pain originating in the extremities and radiating inwards to the limbs and other parts of the body, OR
- •3.2. Chronic pain characterised by burning and tingling paraesthesia
- •4. Clinical condition that, in the opinion of the Investigator, requires treatment with enzyme replacement therapy (ERT).
排除标准
- •1. Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2, calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation (2012).
- •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. Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB), or a change of dose in ongoing treatment, in the 4 weeks prior to screening.
- •4. Subject with 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. Currently taking another investigational drug for any condition.
- •6. Carry only known non-pathogenic Fabry mutations.
- •7. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy).
- •8. History of renal dialysis or kidney transplantation.
- •9. History of or current malignancy requiring treatment.
- •10. Severe cardiomyopathy or significant unstable cardiac disease within 6 months prior to screening.
- •11. 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 antigen assay.
- •12. Presence of any medical, emotional, behavioural, or psychological condition that in the judgement of the Investigator could interfere with the subject’s compliance with the requirements of the study.
- •13. Additional Exclusion Criteria for Subjects Enrolled in Stage I:For subjects enrolled in Stage I (targeting up to 9 subjects total) these specific exclusion criteria, in addition to those above, apply: a) Female b) Non-classic form of Fabry disease c) Receipt of treatment for Fabry disease within 6 months prior to screening d) Positive for anti-PRX-102 antibodies at screening
- •14. Additional Exclusion Criteria for Subjects in Stage II: a) Unwilling to discontinue current ERT treatment for Fabry disease at least 14 days, or chaperone therapy at least 3 days, before baseline.
- •15. Additional Exclusion Criteria for Subjects in Stage II: Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and/or unwilling to undergo pregnancy testing as outlined and to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion. Note: 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. A female subject is considered of childbearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating
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