Treatment of severe steroid-refractory acute GvHD with mesenchymal stromal cells. A phase III randomized double-blind multi-center HOVON study.

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 75

Inclusion Criteria

• Grade II-IV acute GvHD with gut and/or liver involvement, confirmed by histology of involved tissues (in case of gut and liver involvement histology of either one of these tissues is considered sufficient);
• Steroid-refractory defined as progressive disease, mixed response, or grade IV disease after at least 5 days, or stable grade II-III disease after at least 7 days of consecutive systemic treatment with steroids at a dose of >= 2 mg/kg prednisolone or steroid equivalent and a calcineurin-inhibitor at therapeutic trough levels;
• Any age;
• Lansky / Karnofsky score of >=20;
• Signed informed consent by the patient and/or parent(s) or legal guardian(s).

Exclusion Criteria

• Use of intravenous prophylactic MMF <= 6 days prior to development of acute GvHD;
• Systemic treatment for acute GvHD other than steroids and a calcineurin inhibitor (budesonide is considered a local treatment);
• Consecutive treatment with steroids >= 2 mg/kg prednisolone or steroid equivalent > 10 days directly prior to inclusion;
• Previous treatment with advanced therapy medicinal products (ATMP) potentially interfering with the endpoints of this study;
• Known progressive or relapsing malignant disease in case of NHL, HL, CLL, MM, and >= 5% blasts in the bone marrow in case of AML, ALL, CML;
• Requiring ventilator or vasopressor support;
• Poor performance not expected to survive 14 days;
• Known uncontrolled hypersensitivity to DMSO;
• History of any other malignancy, unless diagnosed and treated > 5 years ago with curative intent and without recurrence or nonmelanoma skin cancer and/or carcinoma in situ of any type following complete resection.
• Known pregnancy, a positive higly sensitive pregnancy test at screening, or lactation for female patients; unwillingness to practice highly effective means of contraception for both female and male patients of reproductive potential, as described in paragraph 9.4.
• Any psychological, familial, sociological and /or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Proportion of patients responding to treatment of acute GvHD grade II-IV (with<br /><br>gut and/or liver involvement) at day 29</p><br>

Secondary Outcome Measures

Name	Time	Method

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