Autologous Bone Marrow Stem Cells for Chronic Leg Ulcer Treatment in Sickle Cell Disease

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cell implantation for the treatment of leg ulcer in adult patients with sickle cell disease.

Detailed Description
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Implantation of bone marrow mononuclear cells, including endothelial progenitor cells and mesenchymal stromal cells, into leg ulcers has been shown to improve wound healing. In the present study the safety and efficacy of autologous bone marrow mononuclear cells implantation will be investigated in patients with chronic leg ulcers. Forty cases will be enrolled. Improvement in the pain, rate and extent of leg ulcer wound healing as measured by change in wound surface area will be evaluated until one year.

Official Title
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Efficacy and Safety of Autologous Bone Marrow Stem Cells Infusion for Treatment of Chronic Leg Ulcer in Sickle Cell Disease Patients

Conditions
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Chronic Leg Ulcer, Sickle Cell Disease

Intervention / Treatment
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* Biological: Infusion of Autologous Bone Marrow Mononuclear Cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Stable sickle cell disease patients Patient hospitalized into the dermatology unit Patient with an evolutive leg ulcer since more than 1 year No infection at the time of surgery Patient competent to give informed consent Exclusion Criteria: Patients with a history of corticosteroids or on active therapy infection at the limb affected by ulcer Recurrent painful crises, Immunosuppressive drug therapy, Pregnancy, Presence of neoplastic disease or any other clinical concurrent condition other than sickle cell disease that predisposed them to the development of leg ulcer

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: Control<br>Conventional treatment established by the good clinical practice Patients received standard local care dressing method (compresses) to heal leg ulcers | |
| Experimental: Stem Cell Injection<br>Intramuscular implantation of Autologous bone marrow-derived mononuclear cells | Biological: Infusion of Autologous Bone Marrow Mononuclear Cells<br>* Autologous bone marrow-derived mononuclear cells will be administered by intramuscular injection into and around the leg ulcer. The number of injected cells will be from 5x108 to 1x109 total number of cells on Study Day 1.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of Treatment - Determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events | Determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events | 6 months | 
| Change in Leg Ulcer | Rate and extent of leg ulcer wound healing as measured by change in wound surface area Ulcer diameter will be recorded | 6 months to one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Numeric pain intensity scale (0-10) | Change in Sickle Cell Disease leg ulcer wound pain scores as measured by a 10-point scale (analogic visual scale) and relative to non-wound site pain scores | 6 months to one year | 
| Ulcer Healing | Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area Ulcer diameter will be recorded | 6 months to one year | 
| Quality of Life | Change in quality of life as well as other indicators of patient comfort and well-being | 6 months to one year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cell Therapy, Sickle cell disease, Autologous implantation, Mesenchymal stromal cell, Edothelial Progenitor cell