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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02448121
• Other study ID #: CAAE 25000039812/2005-99
• Secondary ID: 11738
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• First received: May 15, 2015
• Last updated: November 17, 2017
• Start date: August 2006
• Est. completion date: August 2018

Study information

• Verified date: November 2017
• Source: Federal University of Bahia
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 100
• Est. completion date: August 2018
• Est. primary completion date: July 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Avascular Necrosis of Bone
• Necrosis
• Osteonecrosis
• Sickle Cell Disease

Intervention

Procedure:
• Autologous bone marrow stem cell graft
All patients will undergo autologous bone marrow stem cell implantation into the necrotic lesion area using a minimally invasive technique.

Locations

Country	Name	City	State
n/a

Sponsors (3)

Lead Sponsor	Collaborator
Federal University of Bahia	Hospital Universitário Professor Edgard Santos, Oswaldo Cruz Foundation

References & Publications (4)

Daltro GC, Fortuna V, de Souza ES, Salles MM, Carreira AC, Meyer R, Freire SM, Borojevic R. Efficacy of autologous stem cell-based therapy for osteonecrosis of the femoral head in sickle cell disease: a five-year follow-up study. Stem Cell Res Ther. 2015 — View Citation

Hernigou P, Daltro G, Filippini P, Mukasa MM, Manicom O. Percutaneous implantation of autologous bone marrow osteoprogenitor cells as treatment of bone avascular necrosis related to sickle cell disease. Open Orthop J. 2008 Apr 25;2:62-5. doi: 10.2174/1874325000802010062. — View Citation

Hernigou P, Daltro G, Flouzat-Lachaniette CH, Roussignol X, Poignard A. Septic arthritis in adults with sickle cell disease often is associated with osteomyelitis or osteonecrosis. Clin Orthop Relat Res. 2010 Jun;468(6):1676-81. doi: 10.1007/s11999-009-1149-3. Epub 2009 Nov 3. — View Citation

Hernigou P, Flouzat-Lachaniette CH, Daltro G, Galacteros F. Talar Osteonecrosis Related to Adult Sickle Cell Disease: Natural Evolution from Early to Late Stages. J Bone Joint Surg Am. 2016 Jul 6;98(13):1113-21. doi: 10.2106/JBJS.15.01074. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Functional / Clinical Score of 10 points or more	Standardized and objective scoring system to rate patient's functional abilities	60 months
Secondary	Numeric pain intensity scale (0-10)	VAS scoring system	60 months
Secondary	Disease progression defined as progression to a fractural stage of osteonecrosis	Radiographic signals	60 months
Secondary	Reoperation rate	Need for further surgery or hip replacement	60 months
Secondary	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