- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06144515
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Lea Bensoussan, MSc
- Phone Number: +972 (0)586101291
- Email: lea@minoviatx.com
Study Contact Backup
- Name: Natalie Yivgi Ohana, PhD
- Phone Number: +972 (0)54 5833727
- Email: natalie@minoviatx.com
Study Locations
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Jerusalem, Israel, 9103102
- Recruiting
- Shaare Zedek Medical Center
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Contact:
- Yishai Ofran, MD
- Phone Number: +972 (0)2-5645462
- Email: yofran@szmc.org.il
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Contact:
- Chani Waldenberg
- Phone Number: +972 (0)2-5645462
- Email: chanawal@szmc.org.il
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- Male or female patients of 18 years old and up.
- Suspected or previously diagnosed with Myelodysplastic Syndrome.
- 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.
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome
|
Blood is collected in order to perform in vitro research:
Additional Bone marrow (BM) material will be obtained from the same puncture performed for routine clinical tests: |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Investigate mitochondrial content and function in MDS patient-derived cells
Time Frame: 1 Year
|
assays of mitochondrial content and function on whole bone marrow and HSPCs assays of hematopoietic lineages;
|
1 Year
|
investigate effect of mitochondrial augmentation on MDS patient-derived cells
Time Frame: 1 Year
|
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
|
1 Year
|
Persistence of exogenous mtDNA after mitochondrial augmentation
Time Frame: 1 year
|
Persistence of exogenous mtDNA after in vitro or in vivo culturing
|
1 year
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- MNV-007
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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