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Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis (AST-MOMA)

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ClinicalTrials.gov Identifier: NCT01895244
Recruitment Status : Recruiting
First Posted : July 10, 2013
Last Update Posted : April 18, 2019
Sponsor:
Information provided by (Responsible Party):
Joerg Henes, University Hospital Tuebingen

Tracking Information
First Submitted Date  ICMJE July 1, 2013
First Posted Date  ICMJE July 10, 2013
Last Update Posted Date April 18, 2019
Study Start Date  ICMJE September 2012
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
Overall survival [ Time Frame: 3 years ]
Number of patients that are alive after 3 years
Original Primary Outcome Measures  ICMJE
 (submitted: July 4, 2013)
Overall survival [ Time Frame: 3 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
  • 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 ]
    Number of patients that achieve either improvement of >25% in mRSS or > 10% in FVC or DLCO
Original Secondary Outcome Measures  ICMJE
 (submitted: July 4, 2013)
  • 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
Current Other Pre-specified Outcome Measures
 (submitted: July 11, 2013)
Patient reported outcome [ Time Frame: 3 years ]
Differences in Health Assessment Questionnaire (HAQ)
Original Other Pre-specified Outcome Measures
 (submitted: July 4, 2013)
Patient reported outcome [ Time Frame: 3 years ]
Differences in HAQ, FFBh
 
Descriptive Information
Brief Title  ICMJE Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis
Official Title  ICMJE 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 Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Scleroderma
  • Cardiac Involvement
  • Autologous Stem Cell Transplantation
Intervention  ICMJE Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
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
Study Arms  ICMJE
  • Experimental: Conditioning with CYC/ antithymocyte globulin (ATG)
    Each patient receives stem cell transplantation open label with cluster of differentiation (CD)34 selected stem cells mobilisation and conditioning depending on manifestation If cardiac manifestation: Conditioning with CYC 2 x 50mg + thiotepa 2x5mg + ATG If no cardiac manifestation: Conditioning with 4 x 50mg CYC + ATG
    Intervention: Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
  • Experimental: Conditioning with CYC/Thiotepa/ATG
    In patients with cardiac manifestations as defined in the protocol the conditioning for stem cell transplantation is changed to Cyclophosphamide (CYC), thiotepa and ATG
    Intervention: Drug: Autologous stemcell transplantation with CD (cluster of differentiation) 34 selected stem cells
Publications * Henes JC, Koetter I, Horger M, Schmalzing M, Mueller K, Eick C, Bauer A, Vogel W, Kanz L. Autologous stem cell transplantation with thiotepa-based conditioning in patients with systemic sclerosis and cardiac manifestations. Rheumatology (Oxford). 2014 May;53(5):919-22. doi: 10.1093/rheumatology/ket464. Epub 2014 Jan 22.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 4, 2013)
44
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2022
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Joerg C Henes, MD +4970712982711 joerg.henes@med.uni-tuebingen.de
Contact: Theodoros Xenitidis, MD +4970712982711 theodoros.xenitidis@med.uni-tuebingen.de
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01895244
Other Study ID Numbers  ICMJE AST MOMA
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Joerg Henes, University Hospital Tuebingen
Study Sponsor  ICMJE University Hospital Tuebingen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Joerg C Henes, MD University Hospital Tuebingen, Department of oncology, hematology, rheumatology
PRS Account University Hospital Tuebingen
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP