臨床研究等提出・公開システム

Japanese

Date of registration	Mar. 20, 2019
Last modified on	April. 30, 2021
Trial ID	jRCTs031180397

Scientific Title	A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases
Public Title	A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin (Haplo-NM)

Completion date	July. 23, 2020
Result actual enrolment	6
Baseline Characteristics	Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism
Participant flow	The conventional 3+3 design was implemented, where the sequential assessment of the primary endpoint was conducted for each cohort. New patient enrollment was suspended until the confirmation of primary endpoint assessment of the previous cohort was completed. The six subjects in the first and second cohorts completed the protocol treatment without any event at day 30. This pilot trial was successfully terminated.
Adverse events	No serious/unexpected adverse event
Outcome measures	ix cases received HSCT, all cases achieved engraftment at a median of 14.5 days, and no cases developed severe acute GVHD. All cases had sustained donor chimerism without developing chronic GVHD, except one case. No serious adverse event was observed.
Brief summary	Hematopoietic cell transplantation with PTCy and low-dose ATG from HLA-mismatched related donors was feasible to control GVHD for non-malignant diseases in the children.
Date of posting of results summaries	April. 30, 2021
Date of the first journal publication of results	Aug. 14, 2020
URL hyperlink(s) related to results and publications	https://pubmed.ncbi.nlm.nih.gov/32798657/

Plan to share IPD	No
Plan description	NA
URL link to protocol file(s) with version and date	https://jrct.mhlw.go.jp/latest-detail/jRCTs031180397

Contact for Scientific Queries	Name	Kato Motohiro
	Affiliation	National Center for Child Health and Development
	Address	2-10-1, Okura, Setagaya-ku, Tokyo
	Telephone	+81-3-3416-0181
	E-mail	katom-tky@umin.ac.jp
Contact for Public Queries	Name	Kato Motohiro
	Affiliation	National Center for Child Health and Development
	Address	2-10-1, Okura, Setagaya-ku, Tokyo
	Telephone	+81-3-3416-0181
	E-mail	katom-tky@umin.ac.jp

Recruitment status	Complete

Anticipated date of first enrollment		April. 01, 2017
Actual date of first enrollment		April. 16, 2017
Target sample size		9
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	1) Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism 2) Fanconi anemia is excluded for bone marrow failure. 3) No HLA 5/6 (A, B, DR) or more matched related donor 4) No HLA 6/6 (A, B, DR) matched unrelated donor 5) Transplantation from 2- or more-antigen mismatched related donor 6) Age at transplantation <25 years 7) ECOG Performance status: 0-2 8) Sufficient organ function, as follows; a. T-Bil <= 1.5 mg/dl b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older) c. Ejection fraction 45% or better, and QTfc <0.45 sec 9) Obtained informed consent from a guardian of the patient
	Exclusion Criteria	1. Effusion with grade 2 or worse at CTCAE ver 4.0 2. Uncontrolled infection 3. Severe psychological disorders 4. Pregnant or suspected pregnancy 5. Not eligible for this study at the discretion of the investigator.
	Age Minimum	0age over
	Age Maximum	25age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		Bone marrow failure, immunodeficiency, metabolic error
Intervention(s)		Conditioning regimen with anti-thymocyte globulin. Stem cell transplantation at day 0. Cyclophosphamide at day 3 and 4.
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30
Secondary Outcome(s)		GVHD-free survival at day 100, engraftment at day 30, mortality at day 100, incidence of infectious disease at week 8, time from transplantation to onset of a/cGVHD, time from transplantation to death, adverse event

Primary Sponsor

Primary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support	National Center for Child Health and Development
Secondary Sponsor	Not applicable

Name of Certified Review Board	Certified review board of National Center for Child Health and Development
Address	2-10-1, Okura,Setagaya-ku, Tokyo 157-8535, Japan, Tokyo
Telephone	+81-3-3416-0181
E-Mail	rinken@ncchd.go.jp
Approval Status	Approval
Date of approval	Aug. 30, 2018

Secondary ID(s)	UMIN000026159
Issuing Authority	University Hospital Medical Information Network Center

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
3	April. 30, 2021	(this page)	Changes
2	Dec. 24, 2020	Detail	Changes
1	Mar. 20, 2019	Detail

List of change history