UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000028381 |
|---|---|
| Receipt number | R000032488 |
| Scientific Title | A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis |
| Date of disclosure of the study information | 2017/08/01 |
| Last modified on | 2018/01/10 13:54:28 |
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Basic information
Public title
A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis
Acronym
A clinical trial of autologous stem cell transplantation for severe systemic sclerosis
Scientific Title
A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis
Scientific Title:Acronym
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
| 1st name | |
| Middle name | |
| Last name | Koichi Akashi |
Organization
Kyushu University Graduate school of Medicine
Division name
Department of Medicine and Biosystemic Science
Zip code
Address
3-1-1 Maidashi, Higashi-ku, Fukuoka 812-8582, Japan
TEL
092-642-5230
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 812-8582, Japan
TEL
092-642-5228
Homepage URL
mitoma@intmed1.med.kyushu-u.ac.jp
Sponsor or person
Institute
Department of Clinical Immunology and Rheumatology/Infectious Disease, Kyushu University Graduate school of Medicine
Institute
Department
Personal name
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)
IRB Contact (For public release)
Organization
Address
Tel
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 |
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
Open public recruiting
Date of protocol fixation
| 2017 | Year | 05 | Month | 11 | Day |
Date of IRB
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
Other
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000032488
| Research Plan | |
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| Registered date | File name |
| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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