Myfortic - Treatment for Extensive cGvHD

NCT00298324 | Terminated Phase 3 DMC

• 2 other identifiers: EudraCT 2005-006178-86EBMT-LE-0601
• Study Type: interventional
• Target: 34
• Locations: 8 countries
• Sites: 8

Brief Summary

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.

Conditions

Graft vs Host Disease

Keywords

GvHD; graft versus host disease; extensive; myfortic

Outcome Measures

Primary Outcomes (1)

• To test whether the addition of Myfortic improves the efficacy of prednisone plus cyclosporine for treatment of newly diagnosed chronic GvHD, as defined by the proportion of patients with efficacy success at 1 year after enrollment.

  1 year

Secondary Outcomes (6)

• The hazard rates of efficacy success between the two arms. Loss of donor chimerism or recurrent malignancy before secondary systemic therapy and before discontinuation of all immunosuppressive meds will be treated as competing risks.

  1 year

• efficacy failure, and treatment failure defined as efficacy failure or premature discontinuation of study-drug administration due to toxicity

  1 year

• survival without recurrent malignancy

  1 year

• Overall survival

  1 year

• cumulative incidence of secondary systemic treatment for cGvHD before recurrent malignancy

  1 year

Study Arms (2)

Myfortic

ACTIVE COMPARATOR

Patients in this arm will receive Myfortic + Prednisone + Cyclosporine

Drug: Myfortic

Standard Care/ Placebo

OTHER

In this arm patients will receive Prednisone + Cyclosporine + Placebo or Prednisone + Cyclosporine

Drug: Prednisone and Cyclosporine

Interventions

Myfortic DRUG

1440mg twice daily

Myfortic

Prednisone and Cyclosporine DRUG

Prednisone and Cyclosporine given according to protocol. The drugs are tapered according to patient response

Standard Care/ Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• European Society for Blood and Marrow Transplantation: lead
• Novartis: collaborator

Study Sites (8)

Hopital St. Louis

Paris, 75475, France

Location

University Regensburg

Regensburg, 93042, Germany

Location

Ospedale San Martino

Genova, 16132, Italy

Location

University Hospital

Maastricht, 6202, Netherlands

Location

Hospital Clínico Universitario

Valencia, 46010, Spain

Location

Karolinska University Hospital

Huddinge, 141 86, Sweden

Location

University Hospital

Basel, 4031, Switzerland

Location

University Faculty of Medicine

Ankara, 06260, Turkey (Türkiye)

Location

Related Links

• sponsor's website

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

Mycophenolic Acid; Prednisone; Cyclosporine

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Gérard Socié

  Hôptial St Louis, Paris

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK

Study Record Dates

• First Submitted: March 1, 2006
• First Posted: March 2, 2006
• Study Start: September 1, 2006
• Primary Completion: November 1, 2009
• Study Completion: November 1, 2010
• Last Updated: April 3, 2015

Record last verified: 2015-04

Locations

IT (1); DE (1); CH (1); FR (1); NL (1); SE (1); ES (1); TU (1)