臨床研究等提出・公開システム

Japanese

Date of registration	Mar. 18, 2019
Last modified on	June. 30, 2021
Trial ID	jRCTs031180348

Scientific Title	Effectiveness of Curcumin (an active integrant in turmeric) for Pelizaeus-Merzbacher disease: an exploratory study (Effectiveness of Curcumin for PMD)
Public Title	Effectiveness of Curcumin for PMD (Effectiveness of Curcumin for PMD)

Completion date	Dec. 19, 2019
Result actual enrolment	9
Baseline Characteristics	Inclusion criteria : Patients with genetically confirmed Pelizaeus-Merzbacher disease Study Design : single arm study / open(masking not used) / uncontrolled control / single assignment / treatment purpose Age : 5 y.o. to 20 y.o. Sex : Male
Participant flow	Number of patients enroled : 9 Number of patients completed : 9 Number of patients discontinued : 0
Adverse events	Serious adverse event : 0 Unfavorable changes : 0
Outcome measures	Improvement in clinical evaluat ion scores after 12 months treatment GMFM : Increase = or >3 points: 4 cases (44.4%), changes with +/-2 points: 4 cases (44.4%), decrease = or >-3 points; 1 case (11.1%). PMD-FDS : Increase = or >1 point: 3 cases (33.3%), no change: 4 cases (44.4%), decrease= or >-1 point; 2 case (22.2%). Improvement in head MRI findings after 12 months treatment Improvement in myelination : 1 case (11.1%), no change : 8 cases (88.9%).
Brief summary	Nine patients with Pelizaeus-Merzbacher disease were enroled in this study and all completed the study. No Adverse effected was observed. Evaluation of main outcome measure using GMFM showed improvement in 44.4%, and that using PMD-FDS showed improvement in 33.3%. These findings suggested that Curcumin is partially effective for Pelizaeus-Merzbacher disease.
Date of posting of results summaries	June. 30, 2021
Date of the first journal publication of results
URL hyperlink(s) related to results and publications

Plan to share IPD	No
Plan description	Not applicable
URL link to protocol file(s) with version and date	https://jrct.mhlw.go.jp/latest-detail/jRCTs031180348

Contact for Scientific Queries	Name	Inoue Ken
	Affiliation	National Center of Neurology and Psychiatry
	Address	4-1-1 Ogawahigashi-cho, Kodaira, Tokyo
	Telephone	+81-42-346-1713
	E-mail	kinoue@ncnp.go.jp
Contact for Public Queries	Name	Inoue Ken
	Affiliation	National Center of Neurology and Psychiatry
	Address	4-1-1 Ogawahigashi-cho, Kodaira, Tokyo
	Telephone	+81-42-346-1713
	E-mail	kinoue@ncnp.go.jp

Recruitment status	Complete

Anticipated date of first enrollment		April. 01, 2018
Actual date of first enrollment
Target sample size		10
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	no treatment control/standard of care control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	Patients with genetically confir med Pelizaeus-Merzbacher disease
	Exclusion Criteria	1) Individuals who have other s erious medical problems that is not associated with PMD including hepatic, renal or other dysfunctions. 2) Individuals who cannot orally intake medication (those using tube feeding are acceptable). 3)Individuals who is currently taking anti-coagulants. 4) Individuals who is regularly t aking theracurumin or other curcumin-containing products.
	Age Minimum	5age old
	Age Maximum	20age old
	Gender	Male
Health Condition(s) or Problem(s) Studied		Pelizaeus-Merzbacher disease
Intervention(s)		Low dose curcumin (1mg/kg/day, oral, once a day) for 2 months followed by high dose curcumin (4mg/kg/day, oral, once a day) for 10 months.
Health Condition(s) Keyword		Neurology/Pediatrics
Intervention(s) Keyword
Health Condition(s) Code		D020371
Intervention(s) Code
Primary Outcome(s)		Improvement in clinical evaluat ion scores (PMD-FDS, GMFM) after 12 months treatment.
Secondary Outcome(s)		Improvement in the myelinatio n in the brain by T2 and T1 we ighted images (including FAIR) by MRI examination. Shortening of latency by audit ory brainstem response exami nation. Improvement in general health condition including increase of body weight.

Primary Sponsor

Source of Monetary Support / Secondary Sponsor	National Center of Neurology and Psychiatry
Secondary Sponsor	Not applicable

Source of Monetary Support	National Center of Neurology and Psychiatry
Secondary Sponsor	Not applicable

Name of Certified Review Board	National Center of Neurology and Psychiatry Clinical Research Review Board
Address	4-1-1 Ogawa-Higashi, Kodaira, Tokyo 187-8551, Japan, Tokyo
Telephone	+81-42-341-2712-7828
E-Mail	rinri-jimu@ncnp.go.jp
Approval Status	Approval
Date of approval	Mar. 08, 2019

Secondary ID(s)
Issuing Authority

Countries of Recruitment（Except Japan）	None

History of Changes

No	Publication date
2	June. 30, 2021	(this page)	Changes
1	Mar. 18, 2019	Detail

List of change history