Clinical trial NCT02448121: 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
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
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
Lead sponsor: Federal University of Bahia
Sponsor class: OTHER
Collaborators: name: Hospital Universitário Professor Edgard Santos
name: Oswaldo Cruz Foundation
First posted: 2015-05-19
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Change in Functional / Clinical Score of 10 points or more
timeFrame: 60 months
Secondary outcomes:
measure: Numeric pain intensity scale (0-10)
timeFrame: 60 months
measure: Disease progression defined as progression to a fractural stage of osteonecrosis
timeFrame: 60 months
measure: Reoperation rate
timeFrame: 60 months
measure: Radiological progression
timeFrame: 60 months
Other outcomes:
Locations:
Study officials:
affiliation: Federal University of Bahia
name: GIldasio Daltro, MD
role: STUDY_DIRECTOR
affiliation: Federal University of Bahia
name: Vitor Fortuna, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Federal University of Bahia
name: Roberto Meyer, MD
role: STUDY_CHAIR
affiliation: Federal University of Bahia
name: Radovan Borojevic, PhD
role: STUDY_CHAIR
References:
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.
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.
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.
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.
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.