UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Investigation on the effectiveness of histidine copper treatment of Menkes disease patients
Title of the study (Brief title)	Investigation on the effectiveness of histidine copper treatment of Menkes disease patients
Region	Japan

Condition
Condition	Menkes disease
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	The effect of histidine copper treatment in Menkes' disease patients is said to be different depending on the timing of treatment start and gene mutation. The purpose of this study is to clarify the effect of histidine copper on each symptom of Menkes' disease patients participating in the study. In addition, we aim to contribute to the treatment of subjects and other Menkes' disease patients by clarifying that copper deficiency is prevented / improved by histidine copper administration.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	development
Key secondary outcomes	The presence or absence of spasm

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Weekly,subcutaneous injection of histidine copper (750microgram once) is performed three times per week.It is continued every week until the end of the research during the research period.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Those who have written explanation and consent from patients who want family history of histidine copper treatment in Menkes disease patients
Key exclusion criteria	None
Target sample size	2

Research contact person
Name of lead principal investigator	Tokiko Fukuda
Organization	Hamamatsu University School of Medicine
Division name	Department of Pediatrics
Address	1-20-1 Hamdayama,Higashi-ku,Hamamatsu-city,Shizuoka
TEL	053-435-2638
Email	toki-fkd@hama-med.ac.jp

Public contact
Name of contact person	Tokiko Fukuda
Organization	Hamamatsu University School of Medicine
Division name	Department of Pediatrics
Address	1-20-1 Hamdayama,Higashi-ku,Hamamatsu-city,Shizuoka
TEL	053-435-2638
Homepage URL
Email	toki-fkd@hama-med.ac.jp

Sponsor
Institute	Hamamatsu University School of Medicine
Institute
Department

Funding Source
Organization	Self funding
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2017 Year 12 Month 28 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2014 Year 11 Month 28 Day
Anticipated trial start date	2014 Year 11 Month 28 Day
Last follow-up date	2018 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2017 Year 12 Month 28 Day
Last modified on	2018 Year 03 Month 15 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034969