Safety Study of Calcineurin Inhibitor Free GvHD Prophylaxis in Allogeneic Stem Cell Transplantation

University Hospital Freiburg

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Hematologic Diseases

Treatments: Drug: Everolimus and mycophenolate sodium

Study type: Interventional

Funder types: Other

Identifiers

NCT00856505

00557

Details and patient eligibility

About

In stem cell transplantation as treatment for malignant diseases, calcineurin inhibitors like cyclosporine A are commonly used to prevent tissue destruction (GvHD) by activated donor immune cells. The hypothesis for this study is, that replacing calcineurin inhibitors by everolimus and mycophenolate as GvHD prophylaxis not only reduces toxicity of the treatment but also improves tolerance induction of the donor T cells toward the host, eventually increasing the safety of stem cell transplantation.

Enrollment: 38 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of hematologic malignancies, indicated for allogeneic stem cell transplantation:

• acute myeloid leukemia (AML), in CR1, ≥ CR2, primary refractory, relapse

• chronic myeloid leukemia (CML), in chronic phase, in acceleration or blast crisis

• myelodysplastic syndrome (MDS), RA/RARS (transfusion dependent), RAEB, RAEB-t and CMML

• Lymphoma:

  • plasmocytoma
  • immunocytoma (M. Waldenström)
  • chronic-lymphatic leukemia (CLL)
  • additional low and high grade Non-Hodgkin Lymphoma

• Hodgkins disease

• HLA-matched (HLA-A, -B, -DRB1) related or unrelated donor available

• Signed informed consent

Exclusion criteria

• CNS involvement by underlying disease
• Pulmonary disease with VC < 55%, DLCO < 40%
• Cardiac ejection fraction < 30%, uncontrollable arrhythmia
• Creatinin > 1,5 mg/dl or Creatinin-Clearance < 30 ml/min
• Bilirubin > 2 mg/dl
• Active Hepatitis B or C
• HIV serologic positive
• Pregnancy and lactation
• Pre-menstrual women without medical safe contraception
• Participation on another clinical trial in between 30 days before start or during the study only if the clinical trial interferes with the outcome measures.
• Known allergy to study medication or ingredients of the formulation
• Drug- or alcohol abuse
• Non-compliance

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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38 participants in 1 patient group

Everolimus and mycophenolate sodium

Experimental group

Description:

Combination of experimental immunosuppressants for GvHD prophylaxis

Treatment:

Drug: Everolimus and mycophenolate sodium