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

Primary Purpose

Avascular Necrosis of Bone, Sickle Cell Disease

Status

Unknown status

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

Autologous bone marrow stem cell graft

About this trial

This is an interventional treatment trial for Avascular Necrosis of Bone focused on measuring Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell, Humeral Osteonecrosis, Knee Osteonecrosis, Ankle Osteonecrosis

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous bone marrow stem cell graft

Arm Description

Autologous bone marrow stem cell implantation

Outcomes

Primary Outcome Measures

Change in Functional / Clinical Score of 10 points or more

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

Secondary Outcome Measures

Numeric pain intensity scale (0-10)

VAS scoring system

Disease progression defined as progression to a fractural stage of osteonecrosis

Radiographic signals

Reoperation rate

Need for further surgery or hip replacement

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.

Full Information

NCT ID

NCT02448121

First Posted

May 15, 2015

Last Updated

November 17, 2017

Sponsor

Federal University of Bahia

Collaborators

Hospital Universitário Professor Edgard Santos, Oswaldo Cruz Foundation

1. Study Identification

Unique Protocol Identification Number

NCT02448121

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2017

Overall Recruitment Status

Unknown status

Study Start Date

August 2006 (undefined)

Primary Completion Date

July 2018 (Anticipated)

Study Completion Date

August 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Federal University of Bahia

Collaborators

Hospital Universitário Professor Edgard Santos, Oswaldo Cruz Foundation

4. Oversight

Data Monitoring Committee

5. 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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Autologous bone marrow stem cell graft

Arm Type

Experimental

Arm Description

Autologous bone marrow stem cell implantation

Intervention Type

Procedure

Intervention Name(s)

Autologous bone marrow stem cell graft

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in Functional / Clinical Score of 10 points or more

Description

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

Time Frame

60 months

Secondary Outcome Measure Information:

Title

Numeric pain intensity scale (0-10)

Description

VAS scoring system

Time Frame

60 months

Title

Disease progression defined as progression to a fractural stage of osteonecrosis

Description

Radiographic signals

Time Frame

60 months

Title

Reoperation rate

Description

Need for further surgery or hip replacement

Time Frame

60 months

Title

Radiological progression

Description

Time Frame

60 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

GIldasio Daltro, MD

Organizational Affiliation

Federal University of Bahia

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Vitor Fortuna, PhD

Organizational Affiliation

Federal University of Bahia

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Roberto Meyer, MD

Organizational Affiliation

Federal University of Bahia

Official's Role

Study Chair

First Name & Middle Initial & Last Name & Degree

Radovan Borojevic, PhD

Organizational Affiliation

Federal University of Bahia

Official's Role

Study Chair

12. IPD Sharing Statement

Citations:

PubMed Identifier

19478932

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.

Results Reference

background

PubMed Identifier

19885711

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.

Results Reference

background

PubMed Identifier

27385685

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.

Results Reference

background

PubMed Identifier

26021713

Citation

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.

Results Reference

result

PubMed Identifier

29788942

Citation

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.

Results Reference

derived

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

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