Ignite Creation Date:
2024-05-06 @ 7:49 PM
Last Modification Date:
2024-10-26 @ 3:14 PM
Study NCT ID:
NCT06144515
Status:
RECRUITING
Last Update Posted:
2023-11-30
First Post:
2023-10-29
Brief Title:
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
Sponsor:
Minovia Therapeutics Ltd
Organization:
Minovia Therapeutics Ltd
Study Overview
Official Title:
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
Status:
RECRUITING
Status Verified Date:
2023-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 andor subpopulations of cells eg CD34 isolated from the bone marrow aspiration differentiation of augmented andor non-augmented bone marrow aspiration andor subpopulations of cells into hematopoietic lineages eg erythroid megakaryocyte etc assays of mitochondrial content and function assays of hematopoietic lineages culture of augmented andor non-augmented bone marrow aspiration andor subpopulations cryopreservation of augmented andor non-augmented bone marrow aspiration andor subpopulations sequencing of mitochondrial DNA and nuclear DNA in vivo studies of engraftment biodistribution and function of augmented andor non-augmented bone marrow aspiration andor 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
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 andor subpopulations of cells eg CD34 isolated from the bone marrow aspiration differentiation of augmented andor non-augmented bone marrow aspiration andor subpopulations of cells into hematopoietic lineages eg erythroid megakaryocyte etc assays of mitochondrial content and function assays of hematopoietic lineages culture of augmented andor non-augmented bone marrow aspiration andor subpopulations cryopreservation of augmented andor non-augmented bone marrow aspiration andor subpopulations sequencing of mitochondrial DNA and nuclear DNA in vivo studies of engraftment biodistribution and function of augmented andor non-augmented bone marrow aspiration andor 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None