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NCT Number: NCT01895244

Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis

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.

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Key information

Conditions

Scleroderma Autologous Stem Cell Transplantation Cardiac Involvement Connective Tissue Diseases Scleroderma, Diffuse Scleroderma, Systemic Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University Hospital Tuebingen; Department of oncology, hematology, rheumatology, immunology and pulmology

Tübingen, 72076, Germany

Who can participate

Healthy volunteers accepted: No

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

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/m² 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/µl
  • Thrombopenia < 100,000/µl
  • 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

Treatment and study plan

Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells

Drug

If no active alveolitis: mobilisation with 2x1g Cyclophosphamide If active alveolitis: mobilisation with 2x1.5g Cyclophosphamid If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG

Primary outcomes

  1. Efficay - Overall survival

    Time frame: 3 years

    Number of patients that are alive after 3 years

Secondary outcomes

  1. Safety - Treatment related mortality

    Time frame: 100 days

    Treatment related mortality: number of patients who die during the first 100 days after transplantation

  2. Time to engraftment

    Time frame: 2 months

    Time in days from day 0 to platelet count > 20.000 and granulocytes >500/µl

  3. Progression free survival

    Time frame: 3 years

    Time after transplantation without symptoms of disease activity

  4. Efficacy - Lung function test and Skin

    Time frame: 3 years

    Number of patients that achieve either improvement of >25% in mRSS or > 10% in FVC or DLCO

Other outcomes

  1. Efficacy - Patient reported outcome

    Time frame: 3 years

    Differences in Health Assessment Questionnaire (HAQ)

Sponsors and collaborators

Lead sponsor

University Hospital Tuebingen

Other

Registry information

Official study title

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

Acronym: AST-MOMA

Important dates

Study start
2012
Primary completion
2021
Study completion
2024
First posted
Jul 10, 2013
Registry last updated
Jul 29, 2024

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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