UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis
Title of the study (Brief title)	A clinical trial of autologous stem cell transplantation for severe systemic sclerosis
Region	Japan

Condition
Condition	Severe systemic sclerosis
Classification by specialty	Clinical immunology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To investigate the efficacy of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Pragmatic
Developmental phase	Phase II

Assessment
Primary outcomes	The mean change in modified Rodnan skin score at 12 months after transplantation
Key secondary outcomes	The proportion of 25% improvement in skin score at 12 months The mean change in forced vital capacity at 12 months The mean change in serum KL-6 levels at 12 months Event-free survival Overall survival Incidence of adverse events

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification	NO
Dynamic allocation	NO
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine Device,equipment
Interventions/Control_1	Autologous CD34+ stem cells-selected 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

Eligibility
Age-lower limit	16 years-old <=
Age-upper limit	65 years-old >
Gender	Male and Female
Key inclusion criteria	(1) definite diagnosis of Systemic Sclerosis using 2013 ACR/EULAR SSc criteria (2) between the age of 16 and 65 (3) ECOG Performance Status of 0 to 2 (4) refractory to corticosteroids or immunosuppressive therapy (5) fall under any of the following a) with maximum disease duration of 4 years, minimum modified Rodnan skin score of 15 and organ involvement as follows: 1.interstitial pneumonia diagnosed by Chest Xp or CT and less than 80% in %FVC or %DLCO 2.past history of renal crisis 3.reversible heart failure, arrhythmia or pericardial effusion b) with maximum disease duration of 2 years, minimum modified Rodnan skin score of 20 and erythrocyte sedimentation rate greater than 25 mm in the first hour and/or hemoglobin less than 11 g/dl (6) able to understand and willing to sign the Informed Consent Form
Key exclusion criteria	(1) severe organ dysfunction as following a) severe arrhythmia leading to permanent implantation of a pacemaker or implantable cardioverter defibrillator, refractory chronic heart failure, reduced left ventricular ejection fraction lower than 50% at echocardiogram or severe pulmonary hypertension (mPAP >=30 mmHg) b) severe lung dysfunction (PaO2 <60 mmHg under room air conditions, %FVC <50% or %DLOC <20%) c) severe renal dysfunction (eGFR <40 ml/min or serum Cr >=2 mg/dl) d) d) liver abnormality (baseline AST, ALT or total bilirubin levels are >3 times higher than upper limit of normal) (2) presence of malignancy except for complete remission (3) presence of active infection (4) positive for HBs-Ag, HCV-Ab, HIV-Ab or HTLV1-Ab test (5) cumulative dose of more than 10 g of cyclophosphamide (6) history of anaphylaxis or hypersensitivity to murine IgG or iron-dextran complex (7) pregnancy or lactation (8) judged by the investigator to be inappropriate for study participation
Target sample size	12

Research contact person
Name of lead principal investigator	Koichi Akashi
Organization	Kyushu University Graduate school of Medicine
Division name	Department of Medicine and Biosystemic Science
Address	3-1-1 Maidashi, Higashi-ku, Fukuoka 812-8582, Japan
TEL	092-642-5230
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 812-8582, Japan
TEL	092-642-5228
Homepage URL
Email	mitoma@intmed1.med.kyushu-u.ac.jp

Sponsor
Institute	Department of Clinical Immunology and Rheumatology/Infectious Disease, Kyushu University Graduate school of Medicine
Institute
Department

Funding Source
Organization	Department of Clinical Immunology and Rheumatology/Infectious Disease, Kyushu University Graduate school of Medicine
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization	Japan

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	九州大学病院

Other administrative information
Date of disclosure of the study information	2017 Year 08 Month 01 Day

Progress
Recruitment status	Open public recruiting
Date of protocol fixation	2017 Year 05 Month 11 Day
Anticipated trial start date	2017 Year 09 Month 19 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	2017 Year 07 Month 26 Day
Last modified on	2018 Year 01 Month 10 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000032488