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

NCT02448121 | Unknown Phase 1

• 2 other identifiers: CAAE 25000039812/2005-9911738
• Study Type: interventional
• Target: 100
• Locations: 0 countries
• Sites: N/A

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

Conditions

Avascular Necrosis of Bone; Sickle Cell Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Change in Functional / Clinical Score of 10 points or more

  Standardized and objective scoring system to rate patient's functional abilities

  60 months

Secondary Outcomes (4)

• Numeric pain intensity scale (0-10)

  60 months

• Disease progression defined as progression to a fractural stage of osteonecrosis

  60 months

• Reoperation rate

  60 months

• Radiological progression

  60 months

Study Arms (1)

Autologous bone marrow stem cell graft

EXPERIMENTAL

Autologous bone marrow stem cell implantation

Procedure: Autologous bone marrow stem cell graft

Interventions

Autologous bone marrow stem cell graft PROCEDURE

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

Autologous bone marrow stem cell graft

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Federal University of Bahia: lead
• Hospital Universitário Professor Edgard Santos: collaborator
• Oswaldo Cruz Foundation: collaborator

Related Publications (5)

• 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.

  PMID: 19478932 BACKGROUND

• 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.

  PMID: 19885711 BACKGROUND

• 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.

  PMID: 27385685 BACKGROUND

• 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.

  PMID: 26021713 RESULT

• 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.

  PMID: 29788942 DERIVED

MeSH Terms

Conditions

Osteonecrosis; Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Bone Diseases; Musculoskeletal Diseases; Necrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• GIldasio Daltro, MD

  Federal University of Bahia

  STUDY DIRECTOR

• Vitor Fortuna, PhD

  Federal University of Bahia

  PRINCIPAL INVESTIGATOR

• Roberto Meyer, MD

  Federal University of Bahia

  STUDY CHAIR

• Radovan Borojevic, PhD

  Federal University of Bahia

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PhD

Study Record Dates

• First Submitted: May 15, 2015
• First Posted: May 19, 2015
• Study Start: August 1, 2006
• Primary Completion: July 1, 2018
• Study Completion: August 1, 2018
• Last Updated: November 20, 2017

Record last verified: 2017-11