Treatment of severe acute GVHD after allogeneic hematopoietic stem cell transplantation with steroids versus MSC and steroids. A prospective double-blind placebo-controlled randomized phase III trial

Phase 3

Recruiting

• Conditions: stamceltransplantatie gerelateerde complicatie: Graft versus Host ziekte; Graft versus Host Disease; 10018849

• Registration Number: NL-OMON41587
• Lead Sponsor: HOVO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 140

Inclusion Criteria

- Any age;
- Previously treated with allo-SCT/ DLI;
- Acute GVHD grade II iIV nvolving gut and/or liver,
- WHO performance 0-3 ;
- Negative pregnancy test (if applicable);
- Patients must be willing and capable to use adequate contraception during therapy ;
- Written Informed Consent by the patient and/or parent(s) or legal guardian(s);

Exclusion Criteria

- Patients with active, uncontrolled infection;
- Rapid progressive hematological malignancy;
- Patients pre-treated with prednisolone > 1 mg/kg for GVHD, for more than 72 hours prior to randomization/application of MSC/placebo;
- Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, cancer, etc.)
- Any psychological, familial, sociological and/or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
- Known uncontrolled toxicity for DMSO;

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary<br /><br>- Proportion of patients in each treatment arm who experience a CR-GVHD or<br /><br>PR-GVHD at day 57, without treatment failure (initiation of secondary<br /><br>treatment)</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary<br /><br>- Proportion of patients in each treatment arm who experience a CR-GVHD or<br /><br>PR-GVHD at dindicated timepoints (until 2 years), without treatment failure<br /><br>(initiation of secondary treatment)<br /><br>- Time to CR-GVHD or PR-GVHD<br /><br>- Amount of immune suppression at indicated days<br /><br>- Adverse events<br /><br>- The (immunological) phenotype before and after application of MSC/placebo of<br /><br>responders and non-responders in both groups at different sites (see Appendix E<br /><br>and F)<br /><br>- The immunological genotype of responders and non-responders as well as donors<br /><br>in both groups (see Appendix E and F)<br /><br>- Quality of life<br /><br>- Cost-effectiveness<br /><br>- Relapse of the underlying disease (e.g. hematological malignancy)<br /><br>- Progression-free survival<br /><br>- Incidence and severity of chronic GVHD<br /><br>- Overall survival</p><br>