Discarded Bone Marrow for Hematology Research
ConditionHemoglobinopathies
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorSt. Jude Children's Research Hospital
About this trial
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals. The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant.
Secondary objectives
* To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies (e.g. sickle cell disease (SCD) and β-thalassemia). * To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies. * To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patients of any age receiving orthopedic surgery for clinical management that involve bone marrow containing bone discard.
Homozygous S/S disease or doubly heterozygous for S and β thalassemia who are two years or older are eligible.
HbE-β- thalassemia or homozygous (severe) β-thalassemia. including those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Diagnostic criteria include standard hematological parameters, red cell indices, hemoglobin electrophoresis and quantitative determination of HbF and HbA2.
Disqualifiers
Active, acute manifestations of sickle cell disease including painful crisis, acute chest syndrome, cerebrovascular events or active infection.
Pregnant women will not be eligible for study enrollment
Inability or unwillingness of the research participant or legal guardian/representative to give written informed consent will preclude enrollment on this research protocol.
Platelet count < 150,000/mm^3
Trial population
Participants who meet the Eligibility Criteria.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
50 Participants
are grouped into 1 trial groupGroup A: Orthopedic Surgery Patients
Trial outcomes
Primary outcomes
1
Collection, processing and storage of discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals.
Discarded bone marrow-containing bone samples will be collected for use in research; The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant. Samples may be subject to comprehensive assessment of hematopoietic activity using tissue culture based assays, as well as molecular profiling studies of global transcriptome and epigenomes.
Time frame
After completion of sample collection, approximately 10 yearsSponsors and contacts
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Source ClinicalTrials.gov