臨床研究等提出・公開システム

Date of registration	Dec. 06, 2018
Last modified on	Sept. 13, 2022
Trial ID	jRCT2080224174

Scientific Title	A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with Patisiran
Public Title	ALN-TTR02-006

Contact for Scientific Queries	Name
	Contact Name for Scientific Queries	Alnylam Japan K.K.
	Address
	Phone Number
	E-mail Address	JPTiken@alnylam.com

Contact for Public Queries	Name
	Contact Name for Public Queries	Medpace Japan KK
	Address
	Phone Number
	E-mail Address	RSJapan1@medpace.com

Recruitment Status	completed

Date of First Enrolment		Sept. 02, 2016
Target Sample Size		211
Study Type		Interventional
Study Design	Study Design	Multicenter, open-label study
Study Design	Objective	treatment purpose
Phase		N/A
Key inclusion & exclusion criteria	Inclusion Criteria	Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
	Exclusion Criteria	Any new or uncontrolled condition that could make the patient unsuitable for participation
	Minimum Age	18age old over
	Maximum Age	85age old under
	Gender	Both
Health Condition or Problem Studied		Hereditary transthyretin-mediated amyloidosis(hATTR amyloidosis)
Intervention(s)		investigational material(s) Generic name etc : Patisiran (ALN-TTR02) INN of investigational material : Patisiran Therapeutic category code : 129 Other agents affecting peripheral nervous system Dosage and Administration for Investigational material : 0.3 mg/kg(q3w) IV Infusion control material(s) Generic name etc : - INN of investigational material : - Therapeutic category code : Dosage and Administration for Investigational material : -
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome		safety Safety and tolerability of long-term dosing of patisiran as measured by the proportion of subjects with adverse events leading to discontinuation of study drug 5 years
Secondary Outcome		safety efficacy Changes from baseline in the following items Neurologic impairment score: NIS, mNIS+7, NIS+7 QOL: QOL-DN, EuroQOL(EQ-5D) Others: R-ODS, COMPASS 31, serum TTR level, mBMI, NIS-Weakness(NIS-W), 10-Meter Walk Test and grip strength test

Primary Sponsor	Alnylam Pharmaceuticals, Inc. / Medpace Japan KK
Secondary Sponsor	-

Name of Funding Organization	-
Name of Research Funding	-

Name of IRB	Kumamoto University Hospital - IRB
Address of IRB	1-1-1 Honjo, Chuo-ku, Kumamoto City, Kumamoto, Japan
Phone Number of IRB
E-mail Address of IRB
Status of IRB Review	approved
Date of Approval by IRB	July. 27, 2016

Secondary ID No.	JapicCTI-184235
Name of Other Registries

Countries / Regions of Recruitment	Japan/Asia except Japan/North America/South America/Europe/Oceania

History of Changes

No	Publication date
5	Sept. 13, 2022	(this page)	Changes
4	Dec. 17, 2018	Detail	Changes
3	Dec. 07, 2018	Detail	Changes
2	Dec. 06, 2018	Detail	Changes
1	Dec. 06, 2018	Detail

List of change history