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

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ClinicalTrials.gov Identifier: NCT02448121
Recruitment Status : Active, not recruiting
First Posted : May 19, 2015
Last Update Posted : November 20, 2017
Sponsor:
Collaborators:
Hospital Universitário Professor Edgard Santos
Oswaldo Cruz Foundation
Information provided by (Responsible Party):
Vitor Fortuna, 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

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
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 related topics: Osteonecrosis

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.




Primary Outcome Measures :
  1. 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 :
  1. Numeric pain intensity scale (0-10) [ Time Frame: 60 months ]
    VAS scoring system

  2. Disease progression defined as progression to a fractural stage of osteonecrosis [ Time Frame: 60 months ]
    Radiographic signals

  3. Reoperation rate [ Time Frame: 60 months ]
    Need for further surgery or hip replacement

  4. 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.



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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

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
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

Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Vitor Fortuna, PhD, Federal University of Bahia
ClinicalTrials.gov Identifier: NCT02448121     History of Changes
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:
Anemia, Sickle Cell
Necrosis
Osteonecrosis
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Pathologic Processes
Bone Diseases
Musculoskeletal Diseases