A phase 1 multicentre open label dose-escalation study of unrelated, major histocompatibility (MHC)-unmatched mesenchymals stem cells (MSC) for the treatment of steroid refractory acute graft versus host disease in recipients of allogeneic heamatopoietic stem cell transplants
- Conditions
- Patients who have undergone a allogeneic stem cell transplant who have developed steroid refractory graft versus host diseaseInflammatory and Immune System - Other inflammatory or immune system disordersCancer - Other cancer types
- Registration Number
- ACTRN12607000430437
- Lead Sponsor
- Mater Medical Research Institute
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot yet recruiting
- Sex
- All
- Target Recruitment
- 9
Patient is willing and has received an allogeneic bone marrow transplant for a life-threatening disease
Patient has steroid-refractory graft-versus-host disease following the allogeneic bone marrow transplant
Patient or guardian must furnish written informed consent.
Adequate cardiac function with a left ventricular ejection fraction > 45% of predicted.
Adequate pulmonary function pre-haematopietic stem cell transplant, as defined as no severe or symptomatic restrictive or obstructive lung disease, and pulmonary function testing showing an forced expired volume in one second (FEV1) >50% of predicted and a carbon monoxide diffusin capacity (DLCO) >50% of predicted.
(Children less than 6 years of age must have normal oxygen saturation, in the opinion of the Investigator)
Adequate renal function as defined by a creatinine clearance >40% of normal.
Adequate hepatic function as defined by a total bilirubin < 2x normal except for patients with hepatic dysfunction thought due to graft versus host disease.
Adequate neurological function as defined by no evidence of a severe central or peripheral neurological abnormality.
Adequate immunologic function as defined by no evidence of active infection at the time of the transplant preparative regimen.
Female patients are not pregnant, not breast-feeding and are using adequate birth control technique
Patient must be human immunodeficiency virus (HIV)-1 & 2 antibody sero-negative
Patient must demonstrate ability to be compliant with medical regimen.
Patient has active alcohol or substance abuse within 6 months of study entry.
Patient is enrolled on another investigational agent concurrently.
Patient has any medical condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of the patient.
Patient has had a prior haematopoietic stem cell transplant or solid organ transplant
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Infusional toxicity[- 1 hour, 15 minutes post infusion, +30minutes, + 1 hour, + 2 hours and + 4 hours];Adverse events including infections and recurrence of underlying malignancy[Baseline, Day 1 through to Day 27, Day 28, Day 42, Day 100, +12 months and + 24 months];Ectopic Tissue Formation[Baseline, Day 1 through to Day 27, Day 28, Day 42, Day 100, +12 months and + 24 months]
- Secondary Outcome Measures
Name Time Method Response of steroid refractory acute graft versus host disease to the mesenchymal stem cell infusion[Baseline, Day 1 through to Day 27, Day 28, Day 42, Day 100, +12 months and + 24 months]