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

Phase 1

• Conditions: Hematologic Diseases
• Interventions: Drug: Everolimus and mycophenolate sodium

• Registration Number: NCT00856505
• Lead Sponsor: University Hospital Freiburg

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-03
• Status Verified: 2009-03
• Study First Submitted: 2009-03-04
• Study First Submitted QC: 2009-03-04
• Last Update Submitted: 2009-03-04
• Study First Posted: 2009-03-05
• Last Update Posted: 2009-03-05
• Primary Completion: 2010-03
• Study Completion: 2011-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 38

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Everolimus and mycophenolate sodium	Everolimus and mycophenolate sodium	Combination of experimental immunosuppressants for GvHD prophylaxis

Primary Outcome Measures

Name	Time	Method
Toxicity according to CTCAE v3.0	after 100 days and one year after treatment start

Secondary Outcome Measures

Name	Time	Method
Hematopoietic engraftment	day 30 after stem cell transplantation
Incidence of acute and chronic GvHD	one year after stem cell transplantation
Progression free survival	Day 100 and one year after stem cell transplantation
Overall survival	day 100 and one year after stem cell transplantation

Trial Locations

Locations (1)

University Medical Center, Division Hematology/Oncology; 🇩🇪 Freiburg, Baden-Wuerttemberg, Germany