Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis

  • STATUS
    Recruiting
  • End date
    Sep 26, 2024
  • participants needed
    44
  • sponsor
    University Hospital Tuebingen
Updated on 26 January 2021
cyclophosphamide
pulmonary function test
chest ct
fibrosis
progressive systemic sclerosis
diffuse cutaneous systemic sclerosis
MRSS
alveolitis

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.

Details
Condition Scleroderma, Systemic sclerosis, Cardiac Involvement, Autologous Stem Cell Transplantation, Autologous Hematopoietic Stem Cell Transplantation, progressive systemic sclerosis
Treatment Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
Clinical Study IdentifierNCT01895244
SponsorUniversity Hospital Tuebingen
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Diagnosis of progressive systemic sclerosis <7 years
Progressive course despite cyclophosphamide pretreatment
Cyclophosphamide i.v.: at least 3 x with 500-1000 mg/m every 3-4 weeks or
Cyclophosphamide p.o. with at least 100mg/day for at least 2 months or
Contraindication to treatment with cyclophosphamide
Progress defined as at least one of the following criteria
Increase in the mRSS
Worsening of the lung function
Increase in fibrosis/alveolitis in thorax CT
Worsening kidney function through manifestation of systemic sclerosis
Limited or diffuse cutaneous progressive form of Ssc with organ manifestation in the lungs/heart or kidneys

Exclusion Criteria

Age <18 years
Pregnancy or inadequate contraception
Severe heart failure with ejection fraction (EF) < 30% in echo
Pulmonary arterial hypertension with systolic pulmonary arterial pressure (PAPsys) >50mm Hg
Kidney insufficiency: creatinine clearance <30 ml/min
Reduced lung function
Inspiratory vital capacity (IVC) < 50% of normal
Carbon monoxide (CO)-Diffusion capacity SB < 40%
Previously damaged bone marrow
Leukopenia < 2,000/l
Thrombopenia < 100,000/l
Previous myelotoxic treatment
Cyclophosphamide > 50g cumulative (relative)
Infection (Hepatitis B/C, HIV, Salmonella carrier, syphilis, relative: history of tuberculosis)
Severe concomitant psychiatric illness (depression, psychosis)
Substance dependence
Continued nicotine abuse
Continued alcohol abuse
Continued drug abuse
Consent not given
Poor compliance
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