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

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ClinicalTrials.gov Identifier: NCT02448121

Recruitment Status : Unknown

Verified November 2017 by Vitor Fortuna, Federal University of Bahia.
Recruitment status was: Active, not recruiting

First Posted : May 19, 2015

Last Update Posted : November 20, 2017

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborators:

Hospital Universitário Professor Edgard Santos

Oswaldo Cruz Foundation

Information provided by (Responsible Party):

Vitor Fortuna, Federal University of Bahia

Study Description

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

Condition or disease	Intervention/treatment	Phase
Avascular Necrosis of Bone Sickle Cell Disease	Procedure: Autologous bone marrow stem cell graft	Phase 1 Phase 2

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 Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	100 participants
Allocation:	N/A
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Treatment for Hip, Knee, Ankle and Shoulder Osteonecrosis With Autologous Mononuclear Cells Transplantation in People With Sickle Cell Disease
Study Start Date :	August 2006
Estimated Primary Completion Date :	July 2018
Estimated Study Completion Date :	August 2018

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Sickle cell disease

MedlinePlus related topics: Osteonecrosis

Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: Autologous bone marrow stem cell graft Autologous bone marrow stem cell implantation	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.

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

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

Eligibility Criteria

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Ages Eligible for Study:	18 Years to 55 Years (Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02448121

Sponsors and Collaborators

Federal University of Bahia

Hospital Universitário Professor Edgard Santos

Oswaldo Cruz Foundation

Investigators

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Study Director:	GIldasio Daltro, MD	Federal University of Bahia
Principal Investigator:	Vitor Fortuna, PhD	Federal University of Bahia
Study Chair:	Roberto Meyer, MD	Federal University of Bahia
Study Chair:	Radovan Borojevic, PhD	Federal University of Bahia

More Information

Publications of Results:

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 May 29;6(1):110. doi: 10.1186/s13287-015-0105-2.

Other Publications:

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.

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.

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.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Daltro G, Franco BA, Faleiro TB, Rosario DAV, Daltro PB, Meyer R, Fortuna V. Use of autologous bone marrow stem cell implantation for osteonecrosis of the knee in sickle cell disease: a preliminary report. BMC Musculoskelet Disord. 2018 May 22;19(1):158. doi: 10.1186/s12891-018-2067-x.

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Responsible Party:	Vitor Fortuna, PhD, Federal University of Bahia
ClinicalTrials.gov Identifier:	NCT02448121
Other Study ID Numbers:	CAAE 25000039812/2005-99 11738 ( Registry Identifier: CONEP )
First Posted:	May 19, 2015 Key Record Dates
Last Update Posted:	November 20, 2017
Last Verified:	November 2017

Keywords provided by Vitor Fortuna, Federal University of Bahia:


Cell therapy Sickle cell disease Hip Osteonecrosis Autologous implantation	Mesenchymal stromal cell Humeral Osteonecrosis Knee Osteonecrosis Ankle Osteonecrosis

Additional relevant MeSH terms:

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Osteonecrosis Anemia, Sickle Cell Necrosis Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia	Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Pathologic Processes Bone Diseases Musculoskeletal Diseases

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