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Completed

NCT Number: NCT02516124

EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients

The purpose of this study is 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.

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Key information

Conditions

Autoimmune Diseases Connective Tissue Diseases Immune System Diseases Scleroderma, Systemic Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Badoglio Manuela- EBMT Paris Office

Paris, 75010, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Autologous HSCT

Procedure

1st AHSCT

Primary outcomes

  1. Progression free survival

    Time frame: 2 year post transplant

    Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.

Secondary outcomes

  1. Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix)

    Time frame: 2 year post transplant

    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. Overall Survival

    Time frame: 2 year post transplant

    Overall Survival

  3. Response to treatment

    Time frame: at 1 year post transplant

    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 Capacity for carbon monoxide (DLCO) or Forced Vital Capacity (FVC) as compared to baseline without need of further immunosuppression

  4. Improvement in Quality of life

    Time frame: 2 year post transplant

    Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution

  5. Relapse incidence

    Time frame: 2 year post transplant

    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

  6. 100-day Treatment related mortality

    Time frame: 100 days post transplant

    any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease

Sponsors and collaborators

Lead sponsor

European Society for Blood and Marrow Transplantation

Network

Registry information

Official study 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

Acronym: NISSC

Important dates

Study start
2012
Primary completion
2018
Study completion
2018
First posted
Aug 5, 2015
Registry last updated
May 1, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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