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

• ClinicalTrials.gov Identifier: NCT02448121
• Recruitment Status: Unknown
• First Posted: May 19, 2015
• Last Update Posted: November 20, 2017
• Sponsor: Federal University of Bahia
• Collaborators: Hospital Universitário Professor Edgard Santos; Oswaldo Cruz Foundation
• Information provided by (Responsible Party): Vitor Fortuna, Federal University of Bahia

Tracking Information

• First Submitted Date: May 15, 2015
• First Posted Date: May 19, 2015
• Last Update Posted Date: November 20, 2017
• Study Start Date: August 2006
• Estimated Primary Completion Date: July 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 27, 2017)

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

• Original Primary Outcome Measures (submitted: May 18, 2015): Change in Harris Hip Score of 10 points or more [ Time Frame: 60 months ]

Current Secondary Outcome Measures (submitted: June 27, 2017)

• 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.

Original Secondary Outcome Measures (submitted: May 18, 2015)

• Numeric pain intensity scale (0-10) [ Time Frame: 60 months ]
• Disease progression defined as progression to a fractural stage of osteonecrosis [ Time Frame: 60 months ]
• 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.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis in Sickle Cell Disease
• Official Title: Treatment for Hip, Knee, Ankle and Shoulder Osteonecrosis With Autologous Mononuclear Cells Transplantation in People With Sickle Cell Disease
• 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 Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Avascular Necrosis of Bone
• 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.

Study Arms

Experimental: Autologous bone marrow stem cell graft

Autologous bone marrow stem cell implantation

Intervention: Procedure: Autologous bone marrow stem cell graft

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.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: May 18, 2015): 100
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: August 2018
• Estimated Primary Completion Date: July 2018 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 55 Years (Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT02448121
• Other Study ID Numbers: CAAE 25000039812/2005-99; 11738 ( Registry Identifier: CONEP )
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Vitor Fortuna, Federal University of Bahia
• Original Responsible Party: Same as current
• Current Study Sponsor: Federal University of Bahia
• Original Study Sponsor: Same as current

Collaborators

• Hospital Universitário Professor Edgard Santos
• Oswaldo Cruz Foundation

Investigators

• 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

• PRS Account: Federal University of Bahia
• Verification Date: November 2017