UMIN-CTR Clinical Trial

Public title

Phase 2 trial of autologous hematopoietic stem cell transplantation for severe systemic sclerosis

Acronym

Autologous hematopoietic stem cell transplantation for severe systemic sclerosis

Scientific Title

Phase 2 trial of autologous hematopoietic stem cell transplantation for severe systemic sclerosis

Scientific Title:Acronym

Autologous hematopoietic stem cell transplantation for severe systemic sclerosis

Region

Japan

Condition

Systemic sclerosis

Classification by specialty

Clinical immunology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

Analysis of efficacy of autologous hematopoietic stem cell transplantation for severe systemic sclerosis

Basic objectives2

Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Developmental phase

Phase II

Primary outcomes

Event-free survival at 2 years after transplantation

Key secondary outcomes

Event-free survival at 1, 3 and 5 years after transplantation
Improvement of modified Rodnan total skin thickness score at 1, 3 and 5 years after transplantation
Improvement of %VC at 1, 3 and 5 years after transplantation
Safety, Progression-free survival, Overall survival

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

Cyclophosphamide 2g/m2, 2 days
Harvest of autologous hematopoietic stem cells
Cyclophosphamide 50mg/kg, 4 days
Transplantation of autologous hematopoietic stem cells

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

65

years-old

>

Gender

Male and Female

Key inclusion criteria

A patient diagnosed as systemic sclerosis with performance status (ECOG) 0 to 2.
a) Within 4 years after onset and modified Rodnan total skin thickness score (mRSS) is 15 or more, and applicable to one of the following organ complication
1) Pulmonary complications: interstitial pneumonia diagnosed by chest X-ray examination or chest CT, and %FVC or %DLCO is less than 80%.
2) Renal complications: systolic blood pressure is 160 mmHg or more or diastolic blood pressure is 110 mmHg or more, urinalysis abnormality (urine protein, urine occult blood, urinary sediment), microangiopathic hemolytic anemia, or elevated serum creatinine
3) cardiac complications: reversible heart failure, or arrhythmia (supraventricular, ventricular, AV block), or pericardial effusion
b) 1) Involvement of sclerosis to the trunk within 2 years after onset, mRSS is 20 or more, and
2) ESR is 25 mm/1h or more and/or Hb is 11 g/dL or less.
c) Progressive interstitial pneumonia during the course even though more than 4 years after onset or lower than 15 mRSS.

Key exclusion criteria

A patient with one of the following severe organ complication,
a) Heart: uncontrollable arrhythmia, uncontrollable heart failure, LVEF is less than 50% in cardiac ultrasound examination, moderate or more pulmonary hypertension (mean pulmonary artery pressure is 40 mmHg or more)
b) Lung: PaO2 is less than 60 mmHg at room air, %VC is less than 50%. %DLCO is less than 20%.
c) Kidney: renal glomerular filtration rate is less than 40 ml/min, serum creatinine is 2 mg/dl or more.
d) Uncontrolled malignant neoplasm
e) Uncontrollable infection.
f) Cyclophosphamide is used more than 10 g in total.

Target sample size

24

Name of lead principal investigator

1st name
Middle name
Last name	Koichi Akashi

Organization

Kyushu University Hospital

Division name

Department of Hematology, Oncology & Cardiovascular medicine, Department of Clinical Immunology and Rheumatology / Infectious Disease

Zip code

Address

3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan

TEL

092-641-1151

Email

akashi@med.kyushu-u.ac.jp

Name of contact person

1st name
Middle name
Last name	Hiroki Mitoma

Organization

Kyushu University Hospital

Division name

Department of Clinical Immunology and Rheumatology / Infectious Disease

Zip code

Address

3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan

TEL

092-641-1151

Homepage URL

Email

mitoma@intmed1.med.kyushu-u.ac.jp

Institute

Kyushu University

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Japanese Governmental office

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

2018

Year

03

Month

28

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

2010

Year

09

Month

06

Day

Date of IRB

Anticipated trial start date

2010

Year

09

Month

07

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2018

Year

03

Month

28

Day

Last modified on

2018

Year

03

Month

28

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036479