Myfortic - Treatment for Extensive cGvHD

European Society for Blood and Marrow Transplantation

Status and phase

Terminated

Phase 3

Conditions

Graft vs Host Disease

Treatments

Drug: Myfortic

Drug: Prednisone and Cyclosporine

Study type

Interventional

Funder types

NETWORK

Industry

Identifiers

NCT00298324

EBMT-LE-0601

EudraCT 2005-006178-86

Details and patient eligibility

About

The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.

Full description

This clinical trial is a European, multi-center, randomized, double blinded placebo-controlled trial comparing CsA+PDN+MPA versus the reference treatment of CsA+PDN alone + placebo, in patients with extensive chronic GvHD. Randomization will be stratified according to:

Platelet number (low versus high risk)
Source of transplantable cells (marrow versus PBSC versus cord blood)

Patients not in progression at 6 weeks post randomization (progression defined as primary failure) will be evaluated for remission (complete or partial) at 3, 6, 9, & 12 months post randomization

Enrollment

34 patients

Sex

All

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Age 18 - 60
Any primary diagnosis requiring treatment by hematopoietic stem cell transplantation
Recipient of a single allogeneic stem cell transplant (bone marrow or peripheral blood stem cells, or cord blood) minimum 80 days ago
Received a graft from a related or an unrelated donor
Conditioning regimen: Myeloablative or non-myeloablative
Patients suffering a first episode of extensive chronic GvHD, without recurrent disease
The diagnosis of chronic GvHD requires the following:
- Distinction from acute GvHD
- Presence of at least one diagnostic clinical sign of chronic GvHD or presence of at least one distinctive sign confirmed by pertinent biopsy or other relevant diagnostic tests
- Exclusion of other possible diagnoses
Receiving a standard prophylaxis regimen for acute GvHD: CsA plus methotrexate, or CSA+MMF for NMA, or a T-cell depleted transplant
Patient gives written informed consent prior to randomization

Exclusion criteria

Patient age less than 18 years or over 60 years.
GvHD prophylaxis by tacrolimus plus methotrexate
Delayed onset acute GvHD following NMA or DLI
Second allogeneic stem cell transplant
Not the first episode of chronic GvHD needing systemic immunosuppressive therapy.
Limited chronic GvHD (Seattle criteria, see Appendix 1)
Uncontrolled systemic infection which in the opinion of the investigator is associated with an increased risk of the patient's death within 1 week of randomization
In the opinion of the investigator, if the patient has significant medical or psychosocial problems or unstable disease status
Pregnant or lactating females
Known hypersensitivity to mycophenolic acid