NCT02516124

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
82

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Dec 2012

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2012

Completed
2.7 years until next milestone

First Submitted

Initial submission to the registry

August 3, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 5, 2015

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2018

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2018

Completed
Last Updated

May 1, 2018

Status Verified

July 1, 2017

Enrollment Period

5.1 years

First QC Date

August 3, 2015

Last Update Submit

April 30, 2018

Conditions

Autoimmune Diseases

Keywords

Autoimmune diseasesSystemic sclerosisAutologous Hematopoietic stem cell transplant

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (6)

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

    2 year post transplant

  • Overall Survival

    2 year post transplant

  • Response to treatment

    at 1 year post transplant

  • Improvement in Quality of life

    2 year post transplant

  • Relapse incidence

    2 year post transplant

  • +1 more secondary outcomes

Study Arms (1)

NISSC

Autologous HSCT

Procedure: Autologous HSCT

Interventions

Autologous HSCTPROCEDURE

1st AHSCT

NISSC

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients treated with autologous HSCT for severe systemic sclerosis

You may qualify if:

  • Autologous HSCT
  • Age between 18 and 65 years at time of transplant.
  • Established diagnosis of progressive systemic sclerosis according to ARA-criteria

You may not qualify if:

  • Pregnancy or inadequate contraception
  • Severe concomitant disease
  • Reduced lung function
  • Previously damaged bone marrow
  • Uncontrolled severe infection
  • Severe concomitant psychiatric illness

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Badoglio Manuela- EBMT Paris Office

Paris, 75010, France

Location

Related Publications (1)

  • Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128.

    PMID: 31949011DERIVED

Related Links

MeSH Terms

Conditions

Autoimmune DiseasesScleroderma, Systemic

Condition Hierarchy (Ancestors)

Immune System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Study Officials

  • Dominique Farge, PhD

    EBMT ADWP

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 3, 2015

First Posted

August 5, 2015

Study Start

December 1, 2012

Primary Completion

January 1, 2018

Study Completion

March 1, 2018

Last Updated

May 1, 2018

Record last verified: 2017-07

Locations

FR(1)