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Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Busulfan, Cyclophosphamide, Horse ATG
Allogeneic stem cell transplant
Sponsored by
St. Jude Children's Research Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring Anemia, Sickle Cell

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria: Diagnosis of severe' disease is denoted by one of the following: Previous central nervous system vaso-occlusive episode with or without residual neurologic findings or Frequent painful vaso-occlusive episodes with significant interference with normal life activities and which necessitates chronic transfusion therapy or Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy. Exclusion criteria: Patient with SCD chronic lung disease greater than or equal to stage 3 Patient with severe renal dysfunction defined as creatinine clearance < 40 ml/min/1.73m2. Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25%. Patient with HIV infection. Pregnant or lactating. Patient with unspecified chronic toxicity that in the opinion of the Principal Investigator is serious enough to detrimentally affect the patient's capacity to tolerate SCT. Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process

Sites / Locations

  • St. Jude Children's Research Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

1

Arm Description

Outcomes

Primary Outcome Measures

To evaluate engraftment, GVHD, hematopoietic and immune reconstitution, and regimen-related mortality and morbidity in patients with severe sickle cell disease undergoing transplant using either HLA matched sibling bone marrow or cord blood grafts.

Secondary Outcome Measures

Full Information

First Posted
September 9, 2005
Last Updated
May 28, 2009
Sponsor
St. Jude Children's Research Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT00186810
Brief Title
Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease
Official Title
Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
May 2009
Overall Recruitment Status
Completed
Study Start Date
December 1992 (undefined)
Primary Completion Date
February 2006 (Actual)
Study Completion Date
October 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
St. Jude Children's Research Hospital

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a genetically matched sibling. The primary aim of the study was to evaluate how well the donated cells migrated to the bone marrow and begin producing healthy red blood cells, white blood cells and platelets (engrafted), how well the recipients immune system recovered, and assess any regimen related toxicities including a potentially life-threatening transplant related complication called graft-versus-host-disease or GVHD.
Detailed Description
The secondary objectives of this protocol evaluated the effect of this transplant procedure on the subsequent clinical course of patients with severe SCD. Specifically, to determine whether pre-transplant organ dysfunction (brain, heart, lung, kidney, liver, spleen, etc) resultant from sickle hemoglobinopathy can be reversed following this particular transplant procedure.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Anemia, Sickle Cell

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Busulfan, Cyclophosphamide, Horse ATG
Intervention Description
Transplant recipients received a myeloablative conditioning regimen of cyclophosphamide, Anti-Thymocyte Globulin (horse), and Busulfan. Cyclosporine and methotrexate were administered for GVHD prophylaxis.
Intervention Type
Procedure
Intervention Name(s)
Allogeneic stem cell transplant
Intervention Description
Allogeneic stem cell transplant Matched sibling donor transplant Cord blood transplant
Primary Outcome Measure Information:
Title
To evaluate engraftment, GVHD, hematopoietic and immune reconstitution, and regimen-related mortality and morbidity in patients with severe sickle cell disease undergoing transplant using either HLA matched sibling bone marrow or cord blood grafts.
Time Frame
March 2007

10. Eligibility

Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria: Diagnosis of severe' disease is denoted by one of the following: Previous central nervous system vaso-occlusive episode with or without residual neurologic findings or Frequent painful vaso-occlusive episodes with significant interference with normal life activities and which necessitates chronic transfusion therapy or Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy. Exclusion criteria: Patient with SCD chronic lung disease greater than or equal to stage 3 Patient with severe renal dysfunction defined as creatinine clearance < 40 ml/min/1.73m2. Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25%. Patient with HIV infection. Pregnant or lactating. Patient with unspecified chronic toxicity that in the opinion of the Principal Investigator is serious enough to detrimentally affect the patient's capacity to tolerate SCT. Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gregory Hale, MD
Organizational Affiliation
St. Jude Children's Research Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
23416852
Citation
Dallas MH, Triplett B, Shook DR, Hartford C, Srinivasan A, Laver J, Ware R, Leung W. Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2013 May;19(5):820-30. doi: 10.1016/j.bbmt.2013.02.010. Epub 2013 Feb 14.
Results Reference
derived
Links:
URL
http://www.stjude.org
Description
St. Jude Children's Research Hospital

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Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

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