UMIN-CTR Clinical Trial

Public title

HLA-haploidentical allogeneic stem cell transplantation for refractory hematopoietic malignancies (OCU13-3)

Acronym

HLA-haploidentical allogeneic stem cell transplantation for refractory hematopoietic malignancies (OCU13-3)

Scientific Title

HLA-haploidentical allogeneic stem cell transplantation for refractory hematopoietic malignancies (OCU13-3)

Scientific Title:Acronym

HLA-haploidentical allogeneic stem cell transplantation for refractory hematopoietic malignancies (OCU13-3)

Region

Japan

Condition

Acute myeloid leukemia(AML)
Acute lymphoblastic leukemia (ALL)
Chronic myeloid leukemia (CML)
Myelodysplastic syndrome (MDS)

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

YES

Narrative objectives1

To assess the safety and efficacy of HLA-haploidentical allogeneic stem cell transplantation from related donor for patients with poor-prognosis or refractory leukemia or myelodysplastic syndrome (MDS) who lack an HLA serological identical related donor.

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Trial characteristics_2

Developmental phase

Phase II

Primary outcomes

Proportion of patients who survive with graft engraftment at 100 days following 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

Fludarabine (15 mg/square meter of body surface area twice a day for 2 days and 30 mg/square meter once a day for 4 days), cytarabine (2 g/square meter twice a day for 2 days), Melphalan (100mg/ square meter per day for 1 day) is used as a conditioning regimen. Cyclophosphamide (25 mg/kg) is given on day 3, 4 after the graft infusion. The donor source is peripheral blood stem cell. Continuous intravenous tacrolimus (0.03 mg/kg/day) and oral mycophenolate mofetil 3,000mg/day are initiated from day 5 after 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

Age-lower limit

15

years-old

<=

Age-upper limit

70

years-old

>

Gender

Male and Female

Key inclusion criteria

1) Patients with poor-prognosis or
refractory leukemia or MDS who lack an
HLA serological identical related donor.
2) Patients who have an HLA
-haploidentical donor of family member
or relative
3) Age >=15 and < 70 years old
4) ECOG PS 0 or 1
5) Normal function of major organs
6) Informed consent has been acquired.

7) Patients who are in need of a prompt
allogeneic transplant
a) De novo AML: refractory to first
induction therapy or relapse after
chemotherapy
b) ALL: refractory to first induction
therapy or relapse after chemotherapy
c) CML in AP or BC: refractory to TKIs
including imatinib, dasatinib and
nilotinib
d) Patients with AML, ALL, CML or MDS
who have relapsed after allogeneic
transplant

8) Patients who have an indication for
allogeneic transplantation due to an
unfavorable prognosis but lack a
suitable related donor
a) Patients with de novo AML in the CR
with an unfavorable chromosome
abnormality including del(5q)/-5,-
7/del(7q), abn 3q, 9q, 11q, 20q, 21q,
17q, t(6;9), t(9;22) or a complex
karyotype
b) Patients with de novo AML in the CR
with normal karyotype and FLT3-ITD
mutation
c) Patients with AML with
intermediate/poor group by JALSG score
d) Patients with ALL in 1CR who have the following poor prognostic factors
i) t(9;22) or t(4;11)
ii) >=35 years of age at diagnosis
iii) WBC count of more than 30,000/uL
for B- ALL, or more than
100,000/uL for T-ALL at diagnosis
e) AML, ALL in the CR state except for
1CR
f) CML in the CR state except for 1CR
g) MDS with RAEB-1, 2, AML with MRC
h) Patients with AML, ALL, or CML in CP
who have relapsed after allogeneic
transplant

Key exclusion criteria

1) Major organ dysfunction
a) Total bilirubin:>= 2.0mg/dl
b) Serum creatinine: >= 2.0mg/dl
c) Ejection fraction: < 50 %
d) Pulmonary function test: %VC <40%,
FEV1.0% <50% or SaO2 <90% on room air
e) AST or ALT >= 3 x UNL

2) Uncontrolled active infection
3) Uncontrolled CNS invasion
4) Poorly controlled insulin-treated
diabetes mellitus
5) Poorly controlled hypertension
6) Patients with a severe complication
including heart failure, coronary
failure, acute myocardial infarction
within the last three months, liver
cirrhosis and interstitial pneumonia
7) Pregnant, lactating or possible
fertile women who may become pregnant
8) Patients with a severe mental
who are likely to be unable to
participate in the study
9) A history of hypersensitivity or
allergy to any drugs in the
conditioning regimen of this
transplant
10) HIV antibody positivity
11) The physician in charge determines
that there is no indication to perform
this intervention.
(Note: HBs antigen positivity and HCV antibody positivity is not exclusion criterion.)

Target sample size

35

Name of lead principal investigator

1st name
Middle name
Last name	Hirohisa Nakamae

Organization

Graduate School of Medicine, Osaka City University

Division name

Hematology

Zip code

Address

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

TEL

06-6645-3881

Email

hirohisa@msic.med.osaka-cu.ac.jp

Name of contact person

1st name
Middle name
Last name	Hirohisa Nakamae

Organization

Graduate School of Medicine, Osaka City University

Division name

Hematology

Zip code

Address

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

TEL

06-6645-3881

Homepage URL

Email

hirohisa@msic.med.osaka-cu.ac.jp

Institute

Osaka City University

Institute

Department

Personal name

Organization

Osaka City University

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

2013

Year

07

Month

26

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

Completed

Date of protocol fixation

2013

Year

07

Month

04

Day

Date of IRB

Anticipated trial start date

2013

Year

08

Month

01

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2013

Year

07

Month

26

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=R000013231