UMIN-CTR Clinical Trial

Public title

HLA haploidentical hematopoietic stem cell transplantation, Phase II trial

Acronym

haplo-SCT (Hyogo), Phase II

Scientific Title

HLA haploidentical hematopoietic stem cell transplantation, Phase II trial

Scientific Title:Acronym

haplo-SCT (Hyogo), Phase II

Region

Japan

Condition

hematologic malignancies, bone marrow failure syndrome, and other hematological disorders which requires hematopoietic stem cell transplantation

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

To evaluate the efficacy of hematopoietic stem cell transplantation from HLA haploidentical family members for hematologic disorders

Basic objectives2

Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Developmental phase

Phase II

Primary outcomes

Overall survival at day 100 after transplantation

Key secondary outcomes

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Historical

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

1

Purpose of intervention

Treatment

Type of intervention

Medicine

Interventions/Control_1

The basic structure of the preconditioning regimen consists of fludarabine (FLU) at 30 mg/m2/day for 6 days (days -9 and -4), melphalan (MEL) at 70 mg/ m2/day for 2 days (days -3 and -2), and rabbit anti-thymocyte globulin (ATG, Thymoglobulin, Sanofi) at 1.25 mg/kg/day for 2 days (days -2 and -1). To ensure engraftment, 3 Gy of total body irradiation (TBI) was added. For active diseases, high-dose cytarabine (CA) at 2 g/m2/day for 4 days (days -9 and -6) was added for the purpose of tumor reduction. To prevent anaphylaxis caused by ATG, methylprednisolone (mPSL) was administered at 2 mg/kg on days -2 and -1. GVHD prophylaxis consists of the continuous infusion of TAC with a target concentration of 10-12 ng/mL started from day -3, and 1 mg/kg/day of mPSL started after day 0. Peripheral blood stem cells (PBSC) were used as a stem cell source, which were infused without being frozen. G-CSF was administered from day 3 to the day of engraftment.

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

Patients with hamatologic disorder who need hematopoietic stem cell transplantation who fulfill the following,

1) age under 70 years old
2) written informed consent
3) no available donor with matched or 1 antigin-mismatched HLA in HLA A, B, and DR.
4) no available donor in BM bank, or no time for waiting the cordination due to urgent transplantation
5) available HLA 2-3 antigen-mismatched donor in family members
6) In a case of high risk diseases, patient can select matched or HLA 1 antigen-mismatched donor (if any) or HLA 2-3 antigen-mismatched donor
7) PS 0-2
8) EF>50%, SpO2>93%, T-Bil<2mg/dl, AST<2.5*institutional upper limit, Cre<1.5*institutional upper limit

Key exclusion criteria

1) allergic for drugs used in this protocol
2) active CNS lesion (exclude original disease)
3) active infection
4) when the physician in chief decides the patient is inappropriate for this protocol

Target sample size

100

Name of lead principal investigator

1st name	Kazuhiro
Middle name
Last name	Ikegame

Organization

Hyogo College of Medicine

Division name

Division of Hematology, Department of Internal Medicine

Zip code

6638501

Address

1-1, Mukogawa-cho, Nishinomiya city, Hyogo

TEL

+81-798-45-6886

Email

haplo@hyo-med.a.cjp

Name of contact person

1st name	Kazuhiro
Middle name
Last name	Ikegame

Organization

Hyogo College of Medicine

Division name

Division of Hematology, Department of Internal Medicine

Zip code

638501

Address

1-1, Mukogawa-cho, Nishinomiya city, Hyogo

TEL

+81-798-45-6886

Homepage URL

Email

kame@hyo-med.a.cjp

Institute

Hyogo College of Medicine

Institute

Department

Personal name

Organization

Ministry of Education, Culture, Science and Technology

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

IRB, Hyogo College of medicine

Address

1-1, Mukogawa-cho, Nishinomiya city, Hyogo

Tel

0798456886

Email

kame@hyo-med.ac.jp

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

2017

Year

06

Month

18

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

Enrolling by invitation

Date of protocol fixation

2017

Year

01

Month

04

Day

Date of IRB

Anticipated trial start date

2017

Year

04

Month

18

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2017

Year

04

Month

18

Day

Last modified on

2019

Year

04

Month

22

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031010