UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Safety and efficacy of thymoglobulin (ATG) for cord blood transplantation for hematologic malignancies
Title of the study (Brief title)	Safety and efficacy of ATG for CBT for hematologic malignancies
Region	Japan

Condition
Condition	Hematologic malignancies with an indication for allogeneic hematopoietic stem cell transplantation (allo- HSCT) Acute myeloid leukemia Myelodysplastic syndrome Chronic myeloid leukemia Malignant lymphoma Acute lymphoblastic leukemia
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To assess the safety and efficacy of thymoglobulin (ATG) containing reduced intensity conditioning for cord blood transplantation for hematological malignancy patients with advanced age or organ dysfunction, who lack a suitable related or unrelated donor, or who require urgent allogeneic stem cell transplantation.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Day 60 survival rate of patients who achieved engraftment 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	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	For conditioning fludarabine 30 mg/square meter for 6 days and melphalan 70mg/square meter for 2 days, and ATG 2.5mg/kg for 3 days are used. GVHD prophylaxis consists of continuous intravenous tacrolimus (0.03mg/kg/day) given from day -1 and oral mycophenolate mofetil given at 2,250mg/day (750mg x three times) from 6h after the completion of transplantation. MMF will be discontinued at day 40 if GVHD is absent. Graft source: HLA -A/B/DR 4/6 alleles or more matched unrelated cord blood. A minimum cell dose required is 2.5 x 10^7/kg (patient's weight)
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	16 years-old <=
Age-upper limit	70 years-old >=
Gender	Male and Female
Key inclusion criteria	Patients with hematologic malignancies which are incurable by only conventional chemotherapy, and therefore has an indication for allogeneic hematopoietic stem cell transplantation. Eligible diseases; (a)AML 1. First CR with high risk 2. Second CR or greater 3. Relapse or failure to achieve CR after the first course of induction chemotherapy (b)MDS 1. Patients with poor prognosis who have IPSS scores of int-2 or high 2. Transfusion dependence requiring RBC transfusion over 2 units per week or platelet transfusion over 10 units per week (c)CML 1. Second CP or greater 2. First CP or tyrosine kinase inhibitor failure (d)Malignant lymphoma 1. Indolent lymphoma First relapse or greater /progression, regardless of sensitivity to prior chemotherapy 2. Agressive lymphoma *First relapse or greater /progression *Clinical response to prior chemotherapy : PR or CR (e)ALL 1. CR (2) Patients lacking a 6/6 or 5/6 HLA antigen-matched related donor (3) Patients lacking an HLA-identical unrelated donor, or patients who require urgent transplantation due to disease status but can hardly receive transplantation from unrelated HLA-matched donor in a timely fashion (4) ECOG performance status score:0-2 (5) Patients with no indication for myeloablative conditioning (6) Signed informed consent Donor eligibility criteria: (1) CB unit preserved in the Japanese cord blood bank (2) HLA-A, B, DR 8/8 allele match or mismatch at one or two alleles. (3) >= 2.5 X 10^7 total nucleated cell /kg (Patient's weight)
Key exclusion criteria	(1)Major organ dysfunction: (a)Ejection fraction: <30% (b)Pulmonary function test: %VC<30%, FEV1.0% <40%, or SaO2 <90% on room air (c)Serum creatinine: >2.0mg/dl (d)Liver function: total bilirubin >2.0mg/dl, AST or ALT >3 x UNL, or patients with chronic active hepatitis or liver cirrhosis (2)Poorly controlled hypertension (3)HIV antibody positivity (4)Uncontrolled active infection (5)Uncontrolled CNS invasion (6)Pregnant, nursing or possibly pregnant woman (7)Patients with severe mental disorder who are likely unable to participate in the study (8)Known hypersensitivity or allergy to any of the drugs in the conditioning regimen of this transplant, or drugs used for GVHD prophylaxis (9)Patients with positive donor-specific HLA antibodies(DSA) (10)Patients with graft failure following allegeneic hematopoietic stem cell transplantation (11)No indication for this study as judged by physician in charge. Note: HBs antigen positivity and HCV antibody positivity is not excluded.
Target sample size	7

Research contact person
Name of lead principal investigator	Masayuki Hino
Organization	Osaka City University, Graduate School of Medicine
Division name	Hematology
Address	1-4-3, Asahi-machi, Abeno-ku, Osaka, Japan. 545-8585
TEL	06-6645-3881
Email	hinom@med.osaka-cu.ac.jp

Public contact
Name of contact person	Mika Nakamae
Organization	Osaka City University, Graduate School of Medicine
Division name	Hematology(Clinical reserch center for hematological malignancies )
Address	1-4-3, Asahi-machi, Abeno-ku, Osaka, Japan. 545-8585
TEL	06-6645-3881
Homepage URL
Email	crc-hematology@med.osaka-cu.ac.jp

Sponsor
Institute	Hematology, Osaka City University, Graduate School of Meicine
Institute
Department

Funding Source
Organization	None
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	2010 Year 09 Month 14 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2010 Year 09 Month 09 Day
Anticipated trial start date	2011 Year 02 Month 02 Day
Last follow-up date
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	2010 Year 09 Month 14 Day
Last modified on	2018 Year 12 Month 05 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000004778