BMT12108, Non-Myeloablative Conditioning and Bone Marrow Transplantation
-
StatusAccepting Candidates
-
Age1 Year - 70 Years
-
SexesAll
-
Healthy VolunteersAccepts Healthy Volunteers
Objective
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.
Details
Full study title | A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Disease and Other Hemoglobinopathies |
Protocol number | OCR20920 |
ClinicalTrials.gov ID | NCT01850108 |
Phase | N/A |
Eligibility
RECIPIENT INCLUSION CRITERIA
Patients who are ineligible for BMT from an HLA-matched sibling donor can proceed to a haplo-BMT. Patients with an HLA-matched related donor will proceed to a matched BMT.
Age 1-70 years
Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)
Patients and donors must be able to sign consent forms. First degree relative should be willing to donate
Patients must be geographically accessible and willing to participate in all stages of treatment.
Eligible diagnoses: Patients with sickle cell anemia such as sickle cell anemia (Hb SS), Hb Sβ° thalassemia, Hb Sβ+thalassemia, Hb SC disease, Hb SE disease, Hb SD disease, Hemoglobin SO* Arab disease HbS with hereditary persistence of fetal hemoglobin. Other significant hemoglobinopathies.
Plus one of the following:
Attenuation of progressive disease (adults):
Severe and debilitating vaso-occlusive pain despite hydroxyurea or regular blood transfusion therapy.
Stroke and silent infarct; stroke or central nervous system event lasting more than 24 hours; MRI changes indicative of brain parenchyma damage and MRA evidence of cerebrovascular disease.
Recurrent acute chest syndrome requiring exchange hospitalization.
Chronic lung disease as defined by progressive restrictive disease irrespective of oxygen requirements.
Chronic kidney disease, CKD stage II * IV
Transfusion dépendent thalassemia
Recipient Exclusion Criteria:
Poor performance status (ECOG>1).
Poor cardiac function: left ventricular ejection fraction
Lead researchers
-
Biljana Horn, MDPediatric Hematologist/Oncologist (Child Cancer Specialist)
-
Jordan Milner, MDPediatric Hematologist/Oncologist (Child Cancer Specialist)
Participate in a study
Here are some general steps to consider when participating in a research study:
-
Step1
Contact the research team
Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.
Primary contact
-
Step2
Get screened to confirm eligibility
You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.
-
Step3
Provide your consent to participate
If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.
-
Step4
Participate
If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.