Clinical Trial: Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Highdose Chemotherapy and Transplantation of 34+ Selected Stem Cell for Progressive Systemic Sclerosis - Modification According to Manifestation

Brief Summary:

Autologous stem cell therapy has been shown to be effective in patients with systemic sclerosis. Nevertheless treatment is associated with treatment related mortality and patients die during follow up despite successful transplantation.

Intention of this trial is to improve overall survival by modifying the existing protocol used for the ASTIS trial.

To reduce treatment toxicity we reduce the dose of Cyclophosphamide (CYC) for mobilisation to 2x1g.

Especially in patients with cardiac manifestations we also modify the conditioning regimen by adding thiotepa and reducing CYC; as CYC has known cardiotoxic side effects.


Detailed Summary:
Sponsor: University Hospital Tuebingen

Current Primary Outcome: Overall survival [ Time Frame: 3 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Treatment related mortality [ Time Frame: 100 days ]
    Treatment related mortality: number of patients who die during the first 100 days after transplantation
  • Time to engraftment [ Time Frame: 2 months ]
    Time in days from day 0 to platelet count > 20.000 and granulocytes >500/µl
  • Progression free survival [ Time Frame: 3 years ]
    Time after transplantation without symptoms of disease activity
  • Efficacy [ Time Frame: 3 years ]
    Reduction of modified Rodnan Skin score (mRSS) after transplantation


Original Secondary Outcome:

  • Treatment related mortality [ Time Frame: 100 days ]
    Treatment related mortality: number of patients who die during the first 100 days after transplantation
  • Time to engraftment [ Time Frame: 2 months ]
    Time in days from day 0 to platelet count > 20.000 and granulocytes >500/µl
  • Progression free survival [ Time Frame: 3 years ]
    Time after transplantation without symptoms of disease activity
  • Efficacy [ Time Frame: 3 years ]
    Reduction of modified Rodnan Skin score after transplantation


Information By: University Hospital Tuebingen

Dates:
Date Received: July 1, 2013
Date Started: September 2012
Date Completion: September 2020
Last Updated: October 24, 2016
Last Verified: October 2016