Status:
COMPLETED
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Lead Sponsor:
Stanford University
Collaborating Sponsors:
University of Minnesota
University of Alabama at Birmingham
Conditions:
Sickle Cell Disease
Thalassemia
Eligibility:
All Genders
1-25 years
Phase:
PHASE2
Brief Summary
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation. T...
Eligibility Criteria
Inclusion
- Patients with sickle cell disease (SCD) 1-25 years of age with an HLA-identical, but unrelated, donor or 1 human leukocyte antigen (HLA) allele mismatched bone marrow or up to 2 HLA antigen mismatched umbilical cord blood (UCB) donor with one or more of the following:
- Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours.
- Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions.
- Recurrent vaso-occlusive pain, 3 or more episodes per year for 3 years or more years; or recurrent priapism.
- Impaired neuropsychological function and/or abnormal cerebral MRI scan or abnormal transcranial Doppler (TCD).
- Stage I or II sickle lung disease.
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate (GFR) 30-50% of the predicted normal value).
- Bilateral proliferative retinopathy and major visual impairment in at least one eye.
- Osteonecrosis of multiple joints with documented destructive changes.
- Requirement for chronic transfusions but with RBC alloimmunization \>2 antibodies during long term transfusion therapy.
- Failure of hydroxyurea (HU) therapy.
- Patients aged 0-21 years with transfusion dependent alpha- or beta-thalassemia who have an HLA-identical or 1 HLA allele mismatched bone marrow or up to 2 HLA mismatched UCB donor.
- Patients aged 0-21 years with Diamond-Blackfan anemia who have an HLA-identical or 1 HLA allele mismatched bone marrow or up to 2 HLA mismatched UCB donor. Diamond- Blackfan anemia patients will only be eligible if they have failed steroid therapy.
Exclusion
- Patients with one or more of the following:
- Karnofsky or Lansky performance score \<70 (See Appendices I and II).
- Stage III-IV lung disease (Appendix III).
- GFR\<30% predicted normal values.
- Pregnant or lactating females.
- Active serious infection whereby patient has been on intravenous antibiotics for one week prior to study entry.
- Any patient with AIDS or HIV seropositivity.
- Any patient with invasive aspergillus infection within one year of study entry.
- Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance.
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2013
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT00957931
Start Date
March 1 2009
End Date
August 1 2013
Last Update
August 6 2018
Active Locations (2)
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1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455