UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000031940
Receipt number R000036479
Scientific Title Phase 2 trial of autologous hematopoietic stem cell transplantation for severe systemic sclerosis
Date of disclosure of the study information 2018/03/28
Last modified on 2018/03/28 09:59:04

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Basic information

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

Condition

Systemic sclerosis

Classification by specialty

Clinical immunology

Classification by malignancy

Others

Genomic information

NO


Objectives

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


Assessment

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


Base

Study type

Interventional


Study design

Basic design

Single arm

Randomization

Non-randomized

Randomization unit


Blinding

Open -no one is blinded

Control

Historical

Stratification


Dynamic allocation


Institution consideration


Blocking


Concealment



Intervention

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



Eligibility

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


Research contact person

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


Public contact

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


Sponsor or person

Institute

Kyushu University

Institute

Department

Personal name



Funding Source

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization



Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization


Address


Tel


Email



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

2018 Year 03 Month 28 Day


Related information

URL releasing protocol


Publication of results

Unpublished


Result

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



Progress

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

Other related information



Management 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


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name