A Phase 2 interventional study of MNV-201 in Mitochondrial Diseases and Pearson Syndrome, sponsored by Minovia Therapeutics Ltd.. Recruiting at 1 site in Israel. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2026-10-13.
Sponsored by Minovia Therapeutics Ltd. · Phase 2, Interventional, and Treatment
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.
189 studies on the registry are indexed under Mitochondrial Diseases; 60 are open to participants now.
This study's planned enrollment of 6 is below the median of 35 across 114 interventional studies indexed under Mitochondrial Diseases.
Browse Mitochondrial Diseases studies →Minovia Therapeutics Ltd. is the lead sponsor of 7 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biological: MNV-201
Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by leukapheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use.
Also known as: CD34+ cells enriched with allogeneic placenta derived mitochondria
Occurrence of treatment-related adverse events
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion
Time frame: 12 months post treatment.
Height SDS
Improvement from baseline to 12 months post treatment in height SDS compared to the calculated change in height SDS in the 12 months prior to treatment.
Time frame: 24 months
Height SDS
Improvement from baseline to 6 months post treatment in height SDS compared to the calculated change in height SDS in the 6 months prior to treatment.
Time frame: 12 months
Calculated GFR Slope
Improvement in calculated GFR slope 6- and/or 12-months post treatment relative to 6 and/or 12 months prior to treatment (respectively)
Time frame: 24 months
Exploratory endpoint: Organ dysfunction
Improvement in any of the measurements below of organ dysfunction at 6- and/or 12-month post-treatment compared to Baseline * Renal glomerulopathy measured by level of cystatin-C or creatinine * Renal tubulopathy measured by urine amino acids * Renal tubulopathy measured by blood bicarbonate * Gastrointestinal activity measured by albumin * Gastrointestinal activity measured by pediatric PedsQL 3.0 Gastrointestinal Symptoms Module * Pancreas activity measured by insulin * Pancreas activity measured by C-peptide * Heart activity measured by ECG * Growth measured by GDF15 * Growth measured by IGF1 * Growth measured by IGFBP * Change in physician organ involvement score (see appendix C) * Renal glomerulopathy measured by slope of change in eGFR calculated from cystatin-C at 6- or 12-month post-treatment compared to that calculated in the 6- or 12- month period prior to baseline.
Time frame: 24 months
Exploratory endpoint: IPMDS
Improvement from baseline in normalized International Pediatric Mitochondrial Disease Scale (IPMDS) scores (total score or each of three separate components) at 6- or 12-month post-treatment compared to Baseline.
Time frame: 12 months
Exploratory endpoint: Frequency of Hospitalizations
Reduction in frequency of hospitalization(s) during the 6- and/or 12-months post treatment relative to the 6 and/or 12 months (respectively) prior to treatment
Time frame: 24 months
Exploratory endpoint: Length of Hospitalizations
Reduction of length of hospitalization(s) during the 6- and/or 12-months post treatment relative to the 6 and/or 12 months (respectively) prior to treatment.
Time frame: 24 months
Exploratory endpoint: exogenous mtDNA
Pharmacokinetic measurements: exogenous mtDNA analysis in PBMCs at 6- and/or 12-month time points
Time frame: 12 months
Exploratory endpoint: Blood based biomarkers
Change in blood-based biomarkers at 6- and/or 12-month post-treatment compared to Baseline * Mitochondrial quantity and activity (full length mtDNA copy number in PBMC and/or whole blood); ATP content of PBMCs; * Anti-mitochondrial antibody level * PBMC subset analysis.
Time frame: 12 months
Exploratory endpoint: Height SDS
Improvement in height SDS at 6 and/or 12 months relative to natural history available (prospective and/or retrospective)
Time frame: 12 months
Exploratory endpoint: weight SDS
Improvement in weight SDS in 6-month and/or 12-month period after treatment relative to 6- and/or 12-month period (respectively) prior to treatment.
Time frame: 24 months
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Minovia Therapeutics Ltd.