Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis 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 cells in sickle cell disease patients with osteonecrosis

Detailed Description
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The aim of this study is to evaluate autologous bone marrow stem cells transplantation as a safe and potentially beneficial treatment for sickle cell disease patients with osteonecrosis.

Official Title
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Treatment for Hip, Knee, Ankle and Shoulder Osteonecrosis With Autologous Mononuclear Cells Transplantation in People With Sickle Cell Disease

Conditions
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Avascular Necrosis of Bone, Sickle Cell Disease

Intervention / Treatment
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* Procedure: Autologous bone marrow stem cell graft


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Stable sickle cell disease patients FICAT classification of osteonecrosis, inclusive of Stage II. Diagnosis will be based on X-Ray and magnetic resonance imaging (MRI). No infection in affected bones at the time of surgery. Patient competent to give informed consent. Scoring at least 20 points on the pain and daily life activities questionnaire. Exclusion Criteria: FICAT stage III or more Patients with a history of corticosteroids or on active therapy Bone infection at the limb affected by necrosis 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 osteonecrosis of the femoral head

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 |
| --- | --- |
| Experimental: Autologous bone marrow stem cell graft<br>Autologous bone marrow stem cell implantation | Procedure: Autologous bone marrow stem cell graft<br>* All patients will undergo autologous bone marrow stem cell implantation into the necrotic lesion area using a minimally invasive technique.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Functional / Clinical Score of 10 points or more | Standardized and objective scoring system to rate patient's functional abilities | 60 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Numeric pain intensity scale (0-10) | VAS scoring system | 60 months | 
| Disease progression defined as progression to a fractural stage of osteonecrosis | Radiographic signals | 60 months | 
| Reoperation rate | Need for further surgery or hip replacement | 60 months | 
| Radiological progression | Radiological progression is defined according to the development of the Ficat- and Steinberg-staging system of osteonecrosis. It is based on radiological findings in X-Ray and MRI scans, respectively, and it is useful in predicting outcomes. | 60 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell, Humeral Osteonecrosis, Knee Osteonecrosis, Ankle Osteonecrosis