UMIN-CTR Clinical Trial

Basic information
Public title	Phase I study of cord blood transplantation with intra-bone marrow injection of mesenchymal stromal cells
Acronym	Phase I study of cord blood transplantation with intra-bone marrow injection of mesenchymal stromal cells
Scientific Title	Phase I study of cord blood transplantation with intra-bone marrow injection of mesenchymal stromal cells
Scientific Title:Acronym	Phase I study of cord blood transplantation with intra-bone marrow injection of mesenchymal stromal cells
Region	Japan

Condition
Condition	Hematological disorder
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the safety of cord blood transplantation with intra-bone marrow injection of mesenchymal stromal cells (MSCs) generated from bone marrow of third-party donor
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Infusional toxicity of MSCs within 14 days after transplantation
Key secondary outcomes

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Maneuver
Interventions/Control_1	Intra-bone marrow injection of MSCs generated from bone marrow of third-party donor 4 hours before cord blood transplantation
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	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1. Hematological disorder eligible for cord blood transplantation 2. Age: 20 years or older 3. Malignant cells in bone marrow: less than 70% 4. Patient's agreement to participate in this study 5. Performance status (ECOG): 0 to 2 6. Cord blood unit: HLA-A, B, DR serotypes 4/6 match, total nuclear cell count at 1.5 x 10e7/kg or higher, prioritize cord blood unit containing more CD34 positive cells 7. Ejection fraction: 40% or higher, %FEV1.0: 60% or higher, %VC: 50% or higher, AST/ALT: less than 5 x ULN, serum creatinine: less than 3 x ULN 8. MSC donor: spouse or relative within the fourth degree of relationship
Key exclusion criteria	1. Positive for HIV-Ab 2. Administration of gemtuzumab ozogamicin within 6 months 3. In pregnancy or breast-feeding 4. Uncontrolled psychiatric disorder 5. Uncontrolled infectious disease 6. Allergy to drugs for transplant preconditioning or GVHD prophylaxis 7. Re-transplantation within 1 year from the preceding transplantation 8. Doctor's decision
Target sample size	5

Research contact person
Name of lead principal investigator	1st name Middle name Last name Hitoshi Kiyoi
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Hematology and Oncology
Zip code
Address	65 Tsurumai-cho, Showa-ku, Nagoya, Aichi, Japan
TEL	052-744-2141
Email	kiyoi@med.nagoya-u.ac.jp

Public contact
Name of contact person	1st name Middle name Last name Makoto Murata
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Hematology and Oncology
Zip code
Address	65 Tsurumai, Showa-ku, Nagoya, Aichi, Japan
TEL	052-744-2145
Homepage URL
Email	mmurata@med.nagoya-u.ac.jp

Sponsor
Institute	Department of Hematology and Oncology, Nagoya University Graduate School of Medicine
Institute
Department

Funding Source
Organization	Department of Hematology and Oncology, Nagoya University Graduate School of Medicine
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization	Japan

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

IRB Contact (For public release)
Organization
Address
Tel
Email

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	名古屋大学医学部附属病院（愛知県）

Other administrative information
Date of disclosure of the study information	2016 Year 12 Month 01 Day

Related information
URL releasing protocol	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5944542/
Publication of results	Published

Result
URL related to results and publications	https://doi.org/10.1002/sctm.20-0381
Number of participants that the trial has enrolled	6
Results	Five adult patients received MSC-CBT, and no adverse events related to intra-BM injection of MSCs were observed. All patients achieved neutrophil, reticulocyte, and platelet recoveries, with median times to recoveries of 21, 35, and 38 days, respectively. Grade II-IV acute GVHD did not develop in MSC-CBT patients. No patients developed chronic GVHD. At 1 year after transplantation, all MSC-CBT patients survived without relapse.
Results date posted	2021 Year 01 Month 08 Day
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics	|
Participant flow	|
Adverse events	|
Outcome measures	|
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2016 Year 11 Month 22 Day
Date of IRB	2016 Year 11 Month 22 Day
Anticipated trial start date	2017 Year 02 Month 25 Day
Last follow-up date	2019 Year 06 Month 08 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded	2020 Year 11 Month 11 Day

Other
Other related information

Management information
Registered date	2016 Year 10 Month 04 Day
Last modified on	2021 Year 01 Month 09 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027681