Autologous Bone Marrow Stem Cells for Chronic Leg Ulcer Treatment in Sickle Cell Disease

Efficacy and Safety of Autologous Bone Marrow Stem Cells Infusion for Treatment of Chronic Leg Ulcer in Sickle Cell Disease Patients

The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cell implantation for the treatment of leg ulcer in adult patients with sickle cell disease.

Study Overview

• Status: Unknown
• Conditions: Sickle Cell Disease; Chronic Leg Ulcer
• Intervention / Treatment: Biological: Infusion of Autologous Bone Marrow Mononuclear Cells

Detailed Description

Implantation of bone marrow mononuclear cells, including endothelial progenitor cells and mesenchymal stromal cells, into leg ulcers has been shown to improve wound healing.

In the present study the safety and efficacy of autologous bone marrow mononuclear cells implantation will be investigated in patients with chronic leg ulcers. Forty cases will be enrolled. Improvement in the pain, rate and extent of leg ulcer wound healing as measured by change in wound surface area will be evaluated until one year.

• Study Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Stable sickle cell disease patients
• Patient hospitalized into the dermatology unit
• Patient with an evolutive leg ulcer since more than 1 year
• No infection at the time of surgery
• Patient competent to give informed consent

Exclusion Criteria:

• Patients with a history of corticosteroids or on active therapy
• infection at the limb affected by ulcer
• 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 leg ulcer

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Control; Conventional treatment established by the good clinical practice Patients received standard local care dressing method (compresses) to heal leg ulcers
Experimental: Stem Cell Injection; Intramuscular implantation of Autologous bone marrow-derived mononuclear cells	Biological: Infusion of Autologous Bone Marrow Mononuclear Cells; Autologous bone marrow-derived mononuclear cells will be administered by intramuscular injection into and around the leg ulcer. The number of injected cells will be from 5x108 to 1x109 total number of cells on Study Day 1.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of Treatment - Determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events	Determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events	6 months
Change in Leg Ulcer	Rate and extent of leg ulcer wound healing as measured by change in wound surface area Ulcer diameter will be recorded	6 months to one year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Numeric pain intensity scale (0-10)	Change in Sickle Cell Disease leg ulcer wound pain scores as measured by a 10-point scale (analogic visual scale) and relative to non-wound site pain scores	6 months to one year
Ulcer Healing	Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area Ulcer diameter will be recorded	6 months to one year
Quality of Life	Change in quality of life as well as other indicators of patient comfort and well-being	6 months to one year

Collaborators and Investigators

• Sponsor: Federal University of Bahia
• Collaborators: Oswaldo Cruz Foundation
• Investigators: Study Chair: José Valber Meneses, MD, Federal University of Bahia; Principal Investigator: Vitor A Fortuna, PhD, Federal University of Bahia

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: August 1, 2006
• Primary Completion (Anticipated): July 1, 2016
• Study Completion (Anticipated): August 1, 2016

Study Registration Dates

• First Submitted: November 30, 2015
• First Submitted That Met QC Criteria: December 1, 2015
• First Posted (Estimate): December 2, 2015

Study Record Updates

• Last Update Posted (Estimate): December 2, 2015
• Last Update Submitted That Met QC Criteria: December 1, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Keywords: Cell Therapy; Sickle cell disease; Mesenchymal stromal cell; Autologous implantation; Edothelial Progenitor cell
• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Ulcer; Anemia, Sickle Cell; Leg Ulcer; Skin Ulcer
• Other Study ID Numbers: 11738 (CONEP)