An observational study in Myelodysplastic Syndromes, sponsored by Minovia Therapeutics Ltd.. Active, not recruiting at 1 site in Israel. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-06-22.
Sponsored by Minovia Therapeutics Ltd. · Observational
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.
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.
1,317 studies on the registry are indexed under Preleukemia; 57 are open to participants now.
This study's planned enrollment of 100 is below the median of 114 across 116 observational studies indexed under Preleukemia.
Browse Preleukemia studies →Minovia Therapeutics Ltd. is the lead sponsor of 7 studies on the registry; 4 are open to participants now.
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Male or female patients of 18 years old and up and suspected or previously diagnosed with Myelodysplastic Syndrome.
Exclusion Criteria:
Procedure: Blood test
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:
Investigate mitochondrial content and function in MDS patient-derived cells
assays of mitochondrial content and function on whole bone marrow and HSPCs assays of hematopoietic lineages;
Time frame: 1 Year
investigate effect of mitochondrial augmentation on MDS patient-derived cells
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., in vitro ability to differentiate to various hematopoeitic lineages, immunophenotyping and single cells studies of cells post-augmentation and post-differentiation
Time frame: 1 Year
Persistence of exogenous mtDNA after mitochondrial augmentation
Persistence of exogenous mtDNA after in vitro or in vivo culturing
Time frame: 1 year
Plan to share: No
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This study is active, not recruiting, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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Minovia Therapeutics Ltd.