UMIN-CTR Clinical Trial

Public title

Safety and efficacy of thymoglobulin (ATG) for cord blood transplantation for hematologic malignancies

Acronym

Safety and efficacy of ATG for CBT for hematologic malignancies

Scientific Title

Safety and efficacy of thymoglobulin (ATG) for cord blood transplantation for hematologic malignancies

Scientific Title:Acronym

Safety and efficacy of ATG for CBT for hematologic malignancies

Region

Japan

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

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

Primary outcomes

Day 60 survival rate of patients who achieved engraftment after transplantation

Key secondary outcomes

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

Name of lead principal investigator

1st name
Middle name
Last name	Masayuki Hino

Organization

Osaka City University, Graduate School of Medicine

Division name

Hematology

Zip code

Address

1-4-3, Asahi-machi, Abeno-ku, Osaka, Japan. 545-8585

TEL

06-6645-3881

Email

hinom@med.osaka-cu.ac.jp

Name of contact person

1st name
Middle name
Last name	Mika Nakamae

Organization

Osaka City University, Graduate School of Medicine

Division name

Hematology(Clinical reserch center for hematological malignancies )

Zip code

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

Institute

Hematology, Osaka City University, Graduate School of Meicine

Institute

Department

Personal name

Organization

None

Organization

Division

Category of Funding Organization

Self funding

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2010

Year

09

Month

14

Day

URL releasing protocol

Publication of results

Unpublished

URL related to results and publications

Number of participants that the trial has enrolled

Results

Results date posted

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

Recruitment status

Terminated

Date of protocol fixation

2010

Year

09

Month

09

Day

Date of IRB

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

Other related information

Registered date

2010

Year

09

Month

14

Day

Last modified on

2018

Year

12

Month

05

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000004778