Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis in Sickle Cell Disease

Phase 1

• Conditions: Avascular Necrosis of Bone; Sickle Cell Disease
• Interventions: Procedure: Autologous bone marrow stem cell graft

• Registration Number: NCT02448121
• Lead Sponsor: Federal University of Bahia

Brief Summary

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

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.

Study Timeline

• Study Start: 2006-08
• Study First Submitted: 2015-05-15
• Study First Submitted QC: 2015-05-18
• Study First Posted: 2015-05-19
• Status Verified: 2017-11
• Last Update Submitted: 2017-11-17
• Last Update Posted: 2017-11-20
• Primary Completion: 2018-07
• Study Completion: 2018-08

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous bone marrow stem cell graft	Autologous bone marrow stem cell graft	Autologous bone marrow stem cell implantation

Primary Outcome Measures

Name	Time	Method
Change in Functional / Clinical Score of 10 points or more	60 months	Standardized and objective scoring system to rate patient's functional abilities

Secondary Outcome Measures

Name	Time	Method
Reoperation rate	60 months	Need for further surgery or hip replacement
Numeric pain intensity scale (0-10)	60 months	VAS scoring system
Disease progression defined as progression to a fractural stage of osteonecrosis	60 months	Radiographic signals
Radiological progression	60 months	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.