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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT01895244
Other study ID # AST MOMA
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date September 2012
Est. completion date September 2024

Study information

Verified date May 2022
Source University Hospital Tuebingen
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 44
Est. completion date September 2024
Est. primary completion date September 2021
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Germany University Hospital Tuebingen; Department of oncology, hematology, rheumatology, immunology and pulmology Tuebingen

Sponsors (1)

Lead Sponsor Collaborator
University Hospital Tuebingen

Country where clinical trial is conducted

Germany, 

Outcome

Type Measure Description Time frame Safety issue
Other Efficacy - Patient reported outcome Differences in Health Assessment Questionnaire (HAQ) 3 years
Primary Efficay - Overall survival Number of patients that are alive after 3 years 3 years
Secondary Safety - Treatment related mortality Treatment related mortality: number of patients who die during the first 100 days after transplantation 100 days
Secondary Time to engraftment Time in days from day 0 to platelet count > 20.000 and granulocytes >500/µl 2 months
Secondary Progression free survival Time after transplantation without symptoms of disease activity 3 years
Secondary Efficacy - Lung function test and Skin Number of patients that achieve either improvement of >25% in mRSS or > 10% in FVC or DLCO 3 years
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