Mitochondrial DiseasesPhase 2NCT06017869
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)
A Phase 2 study testing MNV-201 in people with Mitochondrial Diseases and Pearson Syndrome, run by Minovia Therapeutics Ltd. at 1 site in Israel.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Mitochondrial Diseases, Pearson Syndrome
- What is being tested?
- MNV-201
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteIsrael
- Study dates
- 31 Jul 2023to Dec 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female participants aged from 1 to 18 years old.
- Diagnosis of Pearson Syndrome (current or history) as verified by molecular identification of deletion in mtDNA of peripheral blood. Participants are diagnosed with PS Participant can be in either the PS manifestations of the disease or may have transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history of PS.
- Participants have failure to thrive (height SDS smaller than -1)
- Participants should have at least 12 months' history of body weight and height and calculated GFR (from creatinine) before treatment.
- Body weight ≥ 10 kg.
- Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.
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- Participants' parents or legal guardian have a good understanding of the study and nature of the procedure and are expected to be able to comply with study visit schedules and caregiver assessments without difficulty.
- Participants' parents or legal guardian provides written informed consent prior to study participation.
- Participants are medically able to undergo the study interventions as determined by the Investigator.
Who cannot join
- History of infection with HIV-1, HIV-2, or HTLV I/II.
- Participants have any active infection.
- Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
- Participants are unable to undergo apheresis.
- Participants have known hypersensitivity to murine proteins or iron-dextran.
- Participants have severe chronic infection.
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- Participants have disease or conditions that may risk the participant or interfere with the ability to interpret the study results.
- History of malignancy.
- History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation.
- Participants have had a change in growth hormone regimen in less than 2 years prior to treatment.
- Participants have participated in another clinical trial or received other experimental medications outside a clinical trial within 1 month prior to start of this study.
- Participants who are pregnant or intend to become pregnant in the next 12 months.
- In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Israel1 site, 1 recruiting
- Recruiting
Sheba Medical Center
Ramat Gan, Israel, 5266202
Elad Jacoby, MD, +972 526668355, elad.jacoby@sheba.health.gov.il
Moran Levin, +972523923147, moran.levin@sheba.health.gov.il
About this study
Official title: PHASE II, OPEN LABEL, SINGLE DOSE STUDY OF THE SAFETY AND EFFICACY OF MNV-201 FOR THE TREATMENT OF PEARSON SYNDROME
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
- Mitochondrial Diseases, Pearson Syndrome
- Being tested
- MNV-201
- Keywords
- Autologous, Mitochondrial, Pearson, Transplantation, Stem cell
- Medical subject headings
- Mitochondrial Diseases, VLCAD deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MNV-201Biological
Also called CD34+ cells enriched with allogeneic placenta derived mitochondria
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.
| Study group | Role | What they receive |
|---|---|---|
| Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria | Experimental | Not described Receives: Biological: MNV-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of treatment-related adverse events
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion
Measured: 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.
Measured: 24 months
Secondary outcomes (2)
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.
Measured: 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)
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2023
- Study start31 Jul 2023
- Primary completionDec 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Minovia Therapeutics Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06017869
- Sponsor's protocol ID
- MNV-010
- Status last verified
- Oct 2026
- First submitted
- 24 Aug 2023
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No