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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MNV-201.
- Who it may be relevant to
- Registry conditions: Mitochondrial Diseases, Pearson Syndrome. Basic parameters: 1 year — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Israel
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
PHASE II, OPEN LABEL, SINGLE DOSE STUDY OF THE SAFETY AND EFFICACY OF MNV-201 FOR THE TREATMENT OF PEARSON SYNDROME
Overview
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.
Interventions
- 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.
Primary outcome measures
- Occurrence of treatment-related adverse events [Time frame: 12 months post treatment.]
- Height SDS [Time frame: 24 months]
Secondary outcome measures (2)
- Height SDS [Time frame: 12 months]
- Calculated GFR Slope [Time frame: 24 months]
Eligibility criteria
Inclusion criteria
- 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.
- 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.
Exclusion criteria
- 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.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Israel · 1 center
- Sheba Medical Center — Ramat Gan
Identifiers
NCT: NCT06017869 · MNV-010