Autologous SCT for Progressive Systemic Sclerosis: a Prospective NIS Study

Autoimmune Diseases Clinical Trial

— NISSC  

Official title:

Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis: a Prospective Non-Interventional Approach Across Europe (NISSC) for the Autoimmune Diseases Working Party of the EBMT

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02264405
• Other study ID #: EBMT-8410015
• Status: Recruiting
• Phase: N/A
• First received: September 26, 2014
• Last updated: October 17, 2014
• Start date: December 2012
• Est. completion date: December 2017

Study information

• Verified date: September 2014
• Source: European Group for Blood and Marrow Transplantation
• Contact: Manuela Badoglio
• Phone: +33.1.70.64.24.16
• Email: manuela.badoglio[@]upmc.fr
• Is FDA regulated: No
• Health authority: Germany: Ethics Commission
• Study type: Observational

Clinical Trial Summary

To assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and analysis of routinely collected clinical and biological data.

Description:

Different protocols are used in the different centres, it is not yet clear which approach will be the most efficient and the safest. Every centre will follow its own local protocol for AHSCT which usually refers to the recent update of the EBMT Guidelines for HSCT in autoimmune disease. Patient selection for AHSCT treatment technique with regard to the risk/benefit balance has to be carefully addressed by standard patient pretransplant evaluation, whereas treatment local regimen, follow-ups evaluation, supportive medication and prophylaxis will be recorded and analysed.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: December 2017
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

• Autologous HSCT

• Age between 18 and 65 years at time of transplant.

• Established diagnosis of progressive systemic sclerosis according to ARA-criteria

Exclusion Criteria:

• Pregnancy or inadequate contraception

• Severe concomitant disease

• Reduced lung function

• Previously damaged bone marrow

• Uncontrolled severe infection

• Severe concomitant psychiatric illness

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Autoimmune Diseases
• Scleroderma, Diffuse
• Scleroderma, Systemic

Intervention

Procedure:
• Autologous HSCT
1st AHSCT

Locations

Country	Name	City	State
France	Badoglio Manuela- EBMT Paris Office	Paris

Sponsors (1)

Lead Sponsor	Collaborator
European Group for Blood and Marrow Transplantation

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Progression free survival	Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.	2 year post transplant	No
Secondary	Safety	Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix) Incidence of Adverse Events (AE) and Serious Adverse Events (SE) Neutrophil and platelet engraftment, defined as first day after transplantation with absolute neutrophil count > 500 cells/µL and >20.000 platelets/µL without platelet transfusion, respectively	2 year post transplant	No
Secondary	Overall Survival	Overall Survival	2 year post transplant	No
Secondary	Response to treatment	Response to treatment within 1 year following autologous HSCT, defined as; 25% improvement in mRSS (modified Rodnan Skin Score) and/or; =10% improvement in Diffuse Capacit y for carbon monoxyde (DLCO) or F^rced Vital Capacity (FVC) as compared to baseline without need of further immunosuppression	at 1 year post transplant	No
Secondary	Improvement in Quality of life	Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution	2 year post transplant	No
Secondary	Relapse incidence	Defined as any of the following changes after prior response to treatment on quarterly follow up as defined below: Worsening of mRSS > 25%; New/Worsening of organ manifestation: lungs, heart or kidney	2 year post transplant	No
Secondary	100-day Treatment related mortality	any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease	100 days post transplant	No

External Links

•   Organisation website