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Trial Title:
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
NCT ID:
NCT06144515
Condition:
Myelodysplastic Syndromes
Conditions: Official terms:
Preleukemia
Myelodysplastic Syndromes
Syndrome
Conditions: Keywords:
Mitochondrial
Research
Study type:
Observational
Overall status:
Recruiting
Study design:
Time perspective:
Prospective
Intervention:
Intervention type:
Procedure
Intervention name:
Blood test
Description:
Blood is collected in order to perform in vitro research:
1. If a routine blood test is performed for clinical tests, the sponsor will receive
the results of the Complete blood count (CBC) and an additional blood sample from
the same puncture of 20ml to maximum 40ml will be collected for this study.
2. If no routine blood test is performed for clinical tests, a CBC will be performed
for the study and an additional blood sample of 20ml to maximum 40ml will be
collected.
Additional Bone marrow (BM) material will be obtained from the same puncture performed
for routine clinical tests:
Arm group label:
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
Summary:
The study Objective is to collect samples of bone marrow aspirates and peripheral blood
of patients with MDS for use in non-clinical research to investigate mitochondrial
function sequence and effect of mitochondrial augmentation.
Detailed description:
Mitochondrial dysfunction is often associated with MDS. Studies have shown mitochondrial
DNA (mtDNA) mutations in different MDS subtypes; however, their role in the pathogenesis
and disease progression are not yet clear. Point mutations were found in various
locations in the mitochondrial genome including tRNAs, rRNAs, and mitochondrial proteins.
Mitochondrial fragmentation in hematopoietic stem and progenitor cells (HSPC) can lead to
ineffective hematopoiesis in MDS, suggesting mitochondria as a therapeutic target for
treating MDS.
Mitochondria augmentation therapy (MAT) is a novel cell technology where hematopoietic
stem and progenitor cells (HSPCs) are augmented ex vivo with mitochondria obtained from
donor cells or tissue. MAT is based on the demonstrated ability of isolated mitochondria
to enter cells and impact mitochondrial function and metabolic activity in the recipient
cells. The transfer of mitochondria from cell to cell has been demonstrated using
extracellular vesicles, nanotubes, and micropinocytosis. Mitochondria entering cells
provide copies of normal mtDNA, which can be further propagated via replication within
the recipient cell and via intercellular transfer.
Research will include in vitro and in vivo studies with the bone marrow sample,
including, among other, the following: mitochondrial augmentation of bone marrow
aspiration and/or subpopulations of cells (e.g. CD34+) isolated from the bone marrow
aspiration; differentiation of augmented and/or non-augmented bone marrow aspiration
and/or subpopulations of cells into hematopoietic lineages (e.g. erythroid,
megakaryocyte, etc); assays of mitochondrial content and function; assays of
hematopoietic lineages; culture of augmented and/or non-augmented bone marrow aspiration
and/or subpopulations; cryopreservation of augmented and/or non-augmented bone marrow
aspiration and/or subpopulations; sequencing of mitochondrial DNA and nuclear DNA; in
vivo studies of engraftment, biodistribution and function of augmented and/or
non-augmented bone marrow aspiration and/or subpopulations of cells isolated from the
bone marrow aspiration.
Research will include in vitro studies with the peripheral blood sample, including, among
other, the following: immunophenotyping of peripheral blood cells and immune-related
functional assays; mitochondrial content and function of peripheral blood cells.
Criteria for eligibility:
Study pop:
Male or female patients of 18 years old and up and suspected or previously diagnosed with
Myelodysplastic Syndrome.
Sampling method:
Non-Probability Sample
Criteria:
Inclusion Criteria:
1. Male or female patients of 18 years old and up.
2. Suspected or previously diagnosed with Myelodysplastic Syndrome.
3. Patient able to understand and provide voluntary written informed consent.
Exclusion Criteria:
1. History of prior allogeneic hematopoietic stem cell transplantation, cell therapy,
gene therapy.
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Shaare Zedek Medical Center
Address:
City:
Jerusalem
Zip:
9103102
Country:
Israel
Status:
Recruiting
Contact:
Last name:
Yishai Ofran, MD
Phone:
(0)2-5645462
Phone ext:
+972
Email:
yofran@szmc.org.il
Contact backup:
Last name:
Chani Waldenberg
Phone:
(0)2-5645462
Phone ext:
+972
Email:
chanawal@szmc.org.il
Start date:
February 9, 2023
Completion date:
December 1, 2025
Lead sponsor:
Agency:
Minovia Therapeutics Ltd.
Agency class:
Industry
Source:
Minovia Therapeutics Ltd.
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06144515
