Stem cell transplantation in diffuse cutaneous systemic sclerosis: upfront therapy or rescue therapy after failure of immunosuppressants?

Phase 1

• Conditions: Diffuse cutaneous systemic sclerosis according to the ACR/EULAR criteria; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2019-004718-32-NL
• Lead Sponsor: MC Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-09
• Last Update Posted: 8 April 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

1. Age between 18 and 65 years.
2. Fulfilling the 2013 ACR-EULAR classification criteria
3. Disease duration = 2 years (from onset of first non-Raynaud’s symptoms) and
- progressive skin involvement with a mRSS = 15 (diffuse skin pattern) and/or
- major organ involvement as defined by either:
a) clinically significant respiratory involvement
b) clinically significant renal involvement
c) clinically significant cardiac involvement
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 120
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

A potential subject who meets any of the following criteria will be excluded from participation in this study:
1. Pregnancy or unwillingness to use adequate contraception during study
2. Concomitant severe disease (respiratory,renal, cardiac, liver failure, psychiatric disorders including active drug or alcohol abuse,concurrent neoplasms or myelodysplasis, bone marrow insufficiency, uncontrolled hypertension, uncontrolled acute or chronic infection, including HIV, HTLV-1,2 positivity,ZUBROD-ECOG-WHO Performance Status Scale > 2, known hypersensitivity to any of the study drug constituents)
3. Previous treatments with immunosuppressants > 6 months including mycophenolate mofetil, methotrexate, azathioprine, rituximab, tocilizumab, glucocorticosteroids.
4. Previous treatments with TLI, TBI or alkylating agents including cyclophosphamide.
5. Significant exposure to bleomycin, tainted rapeseed oil, vinyl chloride, trichlorethylene or silica;
6. eosinophilic myalgia syndrome; eosinophilic fasciitis.
7. Poor compliance of the patient as assessed by the referring physicians.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified