UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase 2 trial of autologous hematopoietic stem cell transplantation for severe systemic sclerosis
Title of the study (Brief title)	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	Koichi Akashi
Organization	Kyushu University Hospital
Division name	Department of Hematology, Oncology & Cardiovascular medicine, Department of Clinical Immunology and Rheumatology / Infectious Disease
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	Hiroki Mitoma
Organization	Kyushu University Hospital
Division name	Department of Clinical Immunology and Rheumatology / Infectious Disease
Address	3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL	092-641-1151
Homepage URL
Email	mitoma@intmed1.med.kyushu-u.ac.jp

Sponsor
Institute	Kyushu University
Institute
Department

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)

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

Progress
Recruitment status	Completed
Date of protocol fixation	2010 Year 09 Month 06 Day
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

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
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
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036479