臨床研究等提出・公開システム

Date of registration	June. 08, 2016
Last modified on	Dec. 17, 2018
Trial ID	jRCT2080223236

Scientific Title	Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy
Public Title

Completion Date or Terminated date
Actual Enrolled Number
Baseline Characteristics
Participant flow
Adverse events
Primary Outcome Measures
Secondary Outcome Measures
Brief summary
Posting of journal publication
Date of the first journal publication of results
URL hyperlink(s) related to results and publications

Plan to share IPD
Plan description
URL link to protocol file(s)
Version of protocol file(s)	version: date:

Contact for Scientific Queries	Name
	Contact Name for Scientific Queries
	Address
	Phone Number
	E-mail Address

Contact for Public Queries	Name
	Contact Name for Public Queries	NIPPON SHINYAKU CO., LTD.
	Address	kouhou@mb.nippon-shinyaku.co.jp
	Phone Number
	E-mail Address

Recruitment Status

Date of First Enrolment
Target Sample Size		16
Study Type		Interventional
Study Design	Study Design	Multicenter, Uncontrolled, Open-Label
Study Design	Objective
Phase		1-2
Key inclusion & exclusion criteria	Inclusion Criteria	1) Ambulant or non-ambulant patient 2) Able to treat with skipping of exon 53
	Exclusion Criteria	Has participated in other clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
	Minimum Age	5age old over
	Maximum Age	17age old under
	Gender	Male
Health Condition or Problem Studied		Duchenne muscular dystrophy
Intervention(s)		investigational material(s) Generic name etc : NS-065/NCNP-01 INN of investigational material : Therapeutic category code : 49- Other agents affecting cellular function Dosage and Administration for Investigational material : intravenous infusion
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome		Efficacy
Secondary Outcome		Safety, Pharmacokinetic profile

Primary Sponsor	NIPPON SHINYAKU CO., LTD.
Secondary Sponsor

Name of Funding Organization
Name of Research Funding

Name of IRB
Address of IRB
Phone Number of IRB
E-mail Address of IRB
Status of IRB Review
Date of Approval by IRB

Secondary ID No.	JapicCTI-163291
Name of Other Registries

Countries / Regions of Recruitment

History of Changes

No	Publication date
7	Dec. 17, 2018	(this page)	Changes
6	Aug. 02, 2018	Detail	Changes
5	Aug. 02, 2018	Detail	Changes
4	June. 19, 2017	Detail	Changes
3	June. 19, 2017	Detail	Changes
2	June. 08, 2016	Detail	Changes
1	June. 08, 2016	Detail

List of change history