beta
Trial Radar AI
Clinical Trial NCT06017869 for Mitochondrial Diseases, Pearson Syndrome is recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.
One trial matched filter criteria
Card View
Back to Search

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)

Recruiting
Clinical Trial NCT06017869 is designed to study Treatment for Mitochondrial Diseases, Pearson Syndrome. It is a Phase 2 interventional trial that is recruiting, having started on 31 July 2023, with plans to enroll 6 participants. Led by Minovia Therapeutics Ltd., it is expected to complete by 1 December 2027. The latest data from ClinicalTrials.gov was last updated on 22 June 2025.
Brief 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). 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.

Official Title

PHASE II, OPEN LABEL, SINGLE DOSE STUDY OF THE SAFETY AND EFFICACY OF MNV-201 FOR THE TREATMENT OF PEARSON SYNDROME

Conditions
Mitochondrial DiseasesPearson Syndrome
Other Study IDs
  • MNV-010
NCT ID Number
NCT06017869
Start Date (Actual)
2023-07-31
Last Update Posted
2025-06-22
Completion Date (Estimated)
2027-12
Enrollment (Estimated)
6
Study Type
Interventional
PHASE
Phase 2
Status
Recruiting
Keywords
Autologous
Mitochondrial
Pearson
Transplantation
Stem cell
Primary Purpose
Treatment
Design Allocation
N/A
Interventional Model
Single Group
Masking
None (Open Label)
Arms / Interventions
Participant Group/ArmIntervention/Treatment
ExperimentalAutologous CD34+ cells enriched with allogenic placenta-derived mitochondria
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
Outcome MeasureMeasure DescriptionTime Frame
Occurrence of treatment-related adverse events
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion
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.
24 months
Secondary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
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.
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)
24 months
Eligibility Criteria

Eligible Ages
Child, Adult
Minimum Age
1 Year
Eligible Sexes
All
  1. Male or female participants aged from 1 to 18 years old.
  2. 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.
  3. Participants have failure to thrive (height SDS smaller than -1)
  4. Participants should have at least 12 months' history of body weight and height and calculated GFR (from creatinine) before treatment.
  5. Body weight ≥ 10 kg.
  6. Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.
  7. 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.
  8. Participants' parents or legal guardian provides written informed consent prior to study participation.
  9. Participants are medically able to undergo the study interventions as determined by the Investigator.

  1. History of infection with HIV-1, HIV-2, or HTLV I/II.
  2. Participants have any active infection.
  3. Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
  4. Participants are unable to undergo apheresis.
  5. Participants have known hypersensitivity to murine proteins or iron-dextran.
  6. Participants have severe chronic infection.
  7. Participants have disease or conditions that may risk the participant or interfere with the ability to interpret the study results.
  8. History of malignancy.
  9. History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation.
  10. Participants have had a change in growth hormone regimen in less than 2 years prior to treatment.
  11. 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.
  12. Participants who are pregnant or intend to become pregnant in the next 12 months.
  13. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
Minovia Therapeutics Ltd. logoMinovia Therapeutics Ltd.
Study Central Contact
Contact: Lea Bensoussan, Msc, + 972 586101291, [email protected]
Contact: Natalie Yivgi Ohana, PhD, +972 54 5833727, [email protected]
1 Study Locations in 1 Countries

Israel

Sheba Medical Center, Ramat Gan, Israel, 5266202, Israel
Elad Jacoby, MD, Contact, +972 526668355, [email protected]
Moran Levin, Contact, +972523923147, [email protected]
Recruiting