計劃書編號ION-682884-CS13
試驗執行中
2022-02-01 - 2027-12-31
Phase III
尚未開始3
召募中1
ICD-10G63
歸類於他處疾病所致之多發神經病變
ICD-10G65.0
Guillain-Barré氏症候群後遺症
ICD-10G65.1
其他發炎性多發神經病變後遺症
ICD-10G65.2
毒性多發神經病變後遺症
ICD-9357.4
其他特定疾病所致之多發神經病變
一項開放標示的延伸試驗，評估 ION-682884 用於遺傳性運甲狀腺素蛋白媒介型澱粉樣多發性神經病變患者中的長期安全性和療效
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試驗申請者
保瑞爾生技股份有限公司
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試驗委託 / 贊助單位名稱
保瑞爾生技股份有限公司
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臨床試驗規模
多國多中心
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更新日期
2026/02/01
試驗主持人及試驗醫院
實際收案人數
0 召募中
實際收案人數
0 尚未開始
適應症
治療遺傳性運甲狀腺素蛋白媒介型澱粉樣多發性神經病變
試驗目的
Primary Objective :
The primary objective is to evaluate the safety and tolerability of extended dosing with ION-682884 in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Secondary Objectives :
The secondary objective is to evaluate the efficacy of extended dosing with ION-682884.
藥品名稱
注射液
預充填式注射劑
預充填式注射劑
預充填式注射劑
預充填式注射劑
主成份
ION-682884
劑型
279
230
230
230
230
劑量
150mg/ml
56mg/ml
56mg/ml
評估指標
The primary endpoints (PEP) are change from Baseline (Index Study Baseline and separately open-label extension [OLE] Baseline) in the following measures:
•Platelet count, renal function and transaminases
•Adverse events (AE)
•Use of concomitant medications
•Vital signs and weight
•Physical examination findings
•Clinical laboratory tests
•Electrocardiogram (ECG) parameters
•Thyroid panel tests
•Coagulation tests
•Inflammatory panel tests
•Complement and immunogenicity tests
•Platelet count, renal function and transaminases
•Adverse events (AE)
•Use of concomitant medications
•Vital signs and weight
•Physical examination findings
•Clinical laboratory tests
•Electrocardiogram (ECG) parameters
•Thyroid panel tests
•Coagulation tests
•Inflammatory panel tests
•Complement and immunogenicity tests
主要納入條件
Inclusion Criteria :
1.Satisfactory completion of ION-682884-CS3 as judged by the Investigator and Sponsor, OR diagnosis of hATTR PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (both are Investigator-Sponsored studies with inotersen - the unconjugated version of ION-682884) as judged by the Investigator and Sponsor.
2.Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements
3.Satisfy the following:
a.Females: must be non-pregnant and non-lactating and either:
i.Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy)
ii.Post-menopausal (defined as 12 months of spontaneous amenorrhea in females ＞ 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and FSH levels in the postmenopausal range for the laboratory involved)
iii.Abstinent or
iv.If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884 and agree to receive pregnancy tests per protocol
b.Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the patient’s non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884
4.Willingness to adhere to vitamin A supplementation per protocol
1.Satisfactory completion of ION-682884-CS3 as judged by the Investigator and Sponsor, OR diagnosis of hATTR PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (both are Investigator-Sponsored studies with inotersen - the unconjugated version of ION-682884) as judged by the Investigator and Sponsor.
2.Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements
3.Satisfy the following:
a.Females: must be non-pregnant and non-lactating and either:
i.Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy)
ii.Post-menopausal (defined as 12 months of spontaneous amenorrhea in females ＞ 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and FSH levels in the postmenopausal range for the laboratory involved)
iii.Abstinent or
iv.If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884 and agree to receive pregnancy tests per protocol
b.Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the patient’s non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884
4.Willingness to adhere to vitamin A supplementation per protocol
主要排除條件
Exclusion Criteria :
1.Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study
1.Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study
試驗計畫預計收納受試者人數
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台灣人數
30 人
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全球人數
200 人
