安成生技& Castle Creek Pharma AC-203(CCP-020) Diacerein 1% Ointment 水泡症TFDA (phaseII)

安成生技宣布AC-203獲准進行治療EB第二期臨床試驗 MoneyDJ新聞 2018-02-26 17:29:04 記者 新聞中心 報導 專注於開發抑制發炎體藥品的安成生技(6610)今(26)日宣布已獲得台灣衛生福利部食品藥物管理署(TFDA)核可進行使用AC-203治療先天性表皮分解性水泡症(EB)臨床第二期試驗。該試驗為一項雙盲、個體內比較、概念驗證臨床試驗，將招募病患年齡為2歲以上的EB患者。主要療效指標是減少EB患者皮膚的病灶面積。安成生技預計將於今年4月納入第一名試驗患者。安成表示，雖然公司在亞洲以外地區的合作夥伴Castle Creek Pharmaceuticals(CCP)，正在全球進行使用AC-203(CCP代號:CCP-020)治療單純型先天性表皮分解性水泡症(EBS)的關鍵2/3期臨床試驗(試驗名稱為DLIVERS)，基於AC-203作用機轉的可能潛力和體外試驗的正面成果，公司希望進一步評估AC-203在其他EB類型的安全性和有效性。安成生技總經理兼執行長陳志光則指出，因為根據EB的全球病友組織DEBRA International的資料，雖然大多數EB患者為單純型(EBS，約佔70%)，而失養型(DEB，約佔25%)和接合型(JEB，約佔5%)為相對少數，但其症狀卻比單純型更嚴重，醫療需求也就更為迫切。另外本試驗獲准得以招募含2歲以上的EB年幼患者，對此罕見疾病有非常重要的意義。因EB患者年齡越小，疾病症狀越嚴重，且患者產生慢性併發症甚至死亡的風險也更高。目前CCP執行的樞紐性臨床試驗DELIVERS被批准的患者年齡則為4歲(含)或以上。本身為JEB患者的母親、DEBRA Taiwan創辦人兼理事長鄭色夢表示，目前並沒有任何已被批准的藥物用於治療任一類型的EB。而EB患者的傷口護理非常費心，對患者和照護人員造成非常嚴重的生理及精神負擔。因為EB對患者和他們的照護者來說是一種極為嚴重的疾病，對有EB患者的家庭來說每一天的生活都可能充滿掙扎；能有減少病灶的有效治療方法對於受此疾病影響的人來說將是一大福音，不僅是在身體上，在精神和士氣方面都將有所助益。對於安成生技致力開發罕見疾病EB治療及其選擇在台灣進行臨床試驗的決定感到興奮，期待與安成生技和醫療團隊合作，為全球所有患者及其照護者找到有效的EB治療方法盡一份心力。

An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)

This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or the CCP-020-101 PK study (feeder studies) without major Clinical Safety Report (CSR) reportable protocol deviations and who meet all the inclusion/exclusion criteria will be eligible to complete 2 treatment cycles of CCP-020.Each treatment cycle will consist of 8 weeks on treatment (once-daily, at home study medication applications) followed by 8 weeks off treatment (only Investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens) with a maximum of 2 treatment cycles allowed for up to 52 weeks. At Baseline Visit (corresponding to the final study visit of the feeder study), the investigator will perform a clinical assessment and determine if any of the subject's lesions require treatment (up to 30% BSA). If the subject has active lesions as determined by the Investigator's clinical assessment, the subject will initiate a cycle of once-daily, at home study medication application to their EBS lesions for 8 weeks (treatment period) followed by an 8-week period where no treatment will be administered. Subjects presenting with no active lesions (as determined by the Investigator's clinical assessment) will not begin treatment and instead, will be instructed to return to the clinic for re-evaluation in 8 weeks or upon worsening of EBS lesions, whichever happens first.

Evaluate Safety and Tolerability of Diacerein 1% Ointment in Terms of the Occurrence of Adverse Events in Subjects with EBS [ Time Frame: From Baseline Visit to 16 weeks, not to exceed 52 weeks cyling between treatment and off treatment periods ] The primary objective of this study is to evaluate the long term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101. Safety will be evaluated in terms of the occurrence of Adverse Events. An adverse event is defined as any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and/or unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational medicinal product, whether or not related to the investigational medicinal product. All adverse events, including observed or volunteered problems, complaints, or symptoms, are to be recorded on the appropriate Case Report Form.

Sponsors and Collaborators  Castle Creek Pharmaceuticals, LLC