Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs - NCT00957931-55455(Clinical Trial 305507)
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Minneapolis |
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State:
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MN |
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55455 |
| Conditions: |
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Sickle Cell Disease - Thalassemia - Diamond-Blackfan Anemia |
| Purpose: |
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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. The patients enrolled in this study will be those who lack matched sibling
donors and therefore have no other option but to undergo bone marrow transplantation using
matched but unrelated bone marrow or umbilical cord blood from the national marrow donor
program registry. Since bone marrow transplantation for these disorders using matched
unrelated donors has two major problems i.e. engraftment, or , the process of new marrow
being accepted and allowed to grow in the the patient; and graft-versus-host disease, or the
process where the new marrow "rejects" the host or the patient, this study has been devised
with methods to overcome these two problems and thus make transplantation from unrelated
donors both successful in terms of engraftment and safe in terms of side effects, both acute
and long term.
In order to accomplish these two goals, two important things will be done. Firstly, patients
will get three medicines which are considered reduced intensity because they are not known
to cause the serious organ damage seen with conventional chemotherapy. These medicines,
however, do cause intense immune suppression so these can cause increased infections.
Secondly, in addition to transplantation of bone marrow from unrelated donors, patients will
also transplanted with mesenchymal stromal cells derived from the bone marrow of their
parents. Mesenchymal stromal cells are adult stem cells that are normally found in the bone
marrow and are thought to create the right background for the blood cells to grow. They have
been shown in many animal and human studies to improve engraftment. In addition, they have a
special property by which they prevent and are now even considered to treat graft versus
host disease. Therefore, by using a reduced intensity chemotherapy regimen before transplant
and transplanting mesenchymal stromal cells, we hope to improve engraftment while at the
same time decrease the potential for severe side effects associated with a conventional
transplant which uses extremely high doses of chemotherapy.
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| Study summary: |
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| Criteria: |
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Inclusion Criteria:
- Patients with SCD 1-25 years of age with an HLA-identical, but unrelated, donor or 1
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, 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 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 Criteria:
- 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 BMT with associated strict isolation or
documented history of medical non-compliance. |
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| Study is available at: |
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University of Minnesota
Minneapolis, MN 55455
United States
Primary Contact:
Angela Smith, M.D.
Email: smith719@umn.edu
Phone: 612-626-2778
Secondary Contact:
Sandhya Kharbanda, M.D.
Email: skharbanda@peds.uab.edu
Phone: 205-939-9285 |
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If you are interested in this clinical trial please use the contact information above. If you would like to get additional information about this clinical trial please visit ClinicalTrials.gov.
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| Data Source: |
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ClinicalTrials.gov |
| Date Processed: |
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March 23, 2011 |
Modifications to
this listing: |
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Only selected fields are shown, please use the link
above to view all information about this clinical trial. |
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