Actively Recruiting
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Led by Minovia Therapeutics Ltd. · Updated on 2025-06-22
6
Participants Needed
1
Research Sites
226 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy \[LHON\]), many involve multiple organs. Mitochondrial disorders may present at any age and a frequent feature is the increasing number of organs involved in the course of the disease. Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in Minovia's GMP facility.
CONDITIONS
Official Title
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants aged from 1 to 18 years old
- Diagnosis of Pearson Syndrome (current or history) confirmed by molecular identification of mtDNA deletion
- Failure to thrive with height SDS smaller than -1
- At least 12 months history of body weight, height, and calculated GFR before treatment
- Body weight at least 10 kg
- Living parent(s) or legal guardian(s) able to understand and provide voluntary written informed consent
- Parent(s) or guardian(s) able to comply with study visits and caregiver assessments
- Written informed consent provided prior to participation
- Medically able to undergo study interventions as determined by Investigator
You will not qualify if you...
- History of infection with HIV-1, HIV-2, or HTLV I/II
- Any active infection
- Diagnosis of Myelodysplastic Syndrome confirmed by FISH and/or karyotype
- Unable to undergo apheresis
- Known hypersensitivity to murine proteins or iron-dextran
- Severe chronic infection
- Diseases or conditions risking participant safety or interfering with study results
- History of malignancy
- Prior treatment with gene therapy, allogeneic bone marrow or cord blood transplantation
- Change in growth hormone regimen less than 2 years prior to treatment
- Participation in another clinical trial or receipt of experimental medications within 1 month before study start
- Pregnant or intending pregnancy within 12 months
- Investigator's judgment of unsuitability for study participation
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Sheba Medical Center
Ramat Gan, Israel, Israel, 5266202
Actively Recruiting
Research Team
L
Lea Bensoussan, Msc
CONTACT
N
Natalie Yivgi Ohana, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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