A randomized placebo-controlled trial comparing cyclosporine plus steroids with or without Myfortic® as primary treatment for extensive chronic graft-versus host disease - Myfortic - GvHD

• Conditions: patients with newly diagnosed (first episode) extensive chronic Graft versus Host Disease (cGvHD) post allogeneic transplantation for any primary diagnosis; MedDRA version: 7Level: LLTClassification code 10018651

• Registration Number: EUCTR2005-006178-86-SE
• Lead Sponsor: European group for Blood and Marrow Transplantation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-07-19
• Last Update Posted: 26 June 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 400

Inclusion Criteria

1) Age 18 – 60
2) Any primary diagnosis requiring treatment by hematopoietic stem cell transplantation (HSCT)
3) Recipient of a single allogeneic stem cell transplant (bone marrow or peripheral blood stem cells, or cord blood) minimum 80 days ago
4) Transplant from a related or unrelated donor
5) Conditioning regimen: Myeloablative or non-myeloablative
6) Patients with a 1st episode of extensive chronic GvHD, without recurrent disease 7) The diagnosis of chronic GvHD as defined by the NIH diagnostic and scoring system
8) Receiving a standard prophylaxis regimen for acute GvHD: CsA alone, CsA plus methotrexate or CsA+MMF for NMA, or a T-cell depleted transplantation
9) Negative pregnancy test in females of child-bearing potential – see section 5.4
10) Written informed patient consent prior to randomisation
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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1) Age less than 18 or over 60 years old
2) GvHD prophylaxis by tacrolimus plus methotroxate
3) Delayed onset acute GvHD following non-myeloablative conditioning or donor lymphocyte infusion
4) Second allogeneic stem cell transplant
5) Not the first episode of chronic GvHD needing systemic immunosuppressive therapy
6) Limited chronic GvHD (Seattle criteria)
7) MMF used to prevent or treat GvHD in the previous 4 weeks
8) Neutropenia
9) Uncontrolled systemic infection with an increased risk of the patient’s death within 1 week of randomization
10) If the patient has significant medical or psychosocial problems or unstable disease status
11) Pregnant or lactating females
12) Patients and/or female partners of male patients not using methods of birth control as defined in section 5.4 of Protocol
13) Known hypersensitivity to mycophonolic acid
14) No simultaneous participation in other chronic GvHD treatment trials allowed

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To improve remission rates of chronic GvHD with cyclosporine A+ prednisone+ Myfortic® (CsA+PDN+MPA) combination as compared to the reference treatment with CsA+PDN;Secondary Objective: 1) To spare patients from long-term use of corticosteroids (and of their long-term side effects) <br><br>2) To decrease transplant-related morbidity (infectious and non-infectious) <br><br>3) To study time to cessation of any immunosuppressive therapy <br><br>4) To test prospectively the NIH severity index for chronic GvHD;Primary end point(s): 1) Efficacy success, defined as the response rate (Complete Remission or Partial Remission of chronic GvHD) at 1 year post randomization, with no secondary systemic therapy at any time.<br><br>2) the requirement for secondary systemic therapy at 1 year post randomization<br><br>3) incidence of recurrent malignancy at 1 year post randomization (of the original diagnosis requiring transplantation)