Actively Recruiting
Phase II Open-Label Study of MNV-201 Cell Therapy for Pearson Syndrome Treatment
Led by Minovia Therapeutics Ltd. · Updated on 2025-06-22
6
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Primary Mitochondrial diseases are a group of disorders caused by mutations in genes from nuclear DNA or mitochondrial DNA, affecting one or multiple organs. Pearson Syndrome is one such mitochondrial disease that can present with various symptoms and progresses over time. This Phase II open-label study evaluates the safety and therapeutic effects of MNV-201, a cell therapy developed by Minovia Therapeutics Ltd., designed to treat patients diagnosed with Pearson Syndrome. MNV-201 is made by enriching a participant's own CD34+ hematopoietic stem and progenitor cells with mitochondria derived from donated placental tissue. The study involves a single intravenous infusion of these enriched cells. Participants undergo mobilization and leukapheresis to collect their cells, which are then processed and infused back. The trial monitors treatment effects over months and includes follow-ups up to 24 months after treatment. Participants will be involved in regular assessments including monitoring treatment-related adverse events for 12 months and measuring height standard deviation scores (SDS) at 12 and 24 months, plus kidney function through calculated glomerular filtration rate slope over 24 months. The study includes evaluations of growth and safety, requiring participants to have prior growth and kidney function data for at least 12 months before treatment. Parent or guardian consent and ongoing cooperation with study visits and assessments are essential during the trial period.
CONDITIONS
Brief 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 or history verified by molecular identification of mitochondrial DNA deletion
- Failure to thrive with height standard deviation score smaller than -1
- At least 12 months history of body weight, height, and kidney function data before treatment
- Body weight of at least 10 kilograms
- Living parent(s) or legal guardian(s) able to provide voluntary written informed consent
- Parent(s) or legal guardian(s) able to comply with study visit schedules and assessments
- Written informed consent provided by parent(s) or legal guardian(s) prior to participation
- Medically able to undergo study interventions as determined by the Investigator
You will not qualify if you...
- History of infection with HIV-1, HIV-2, or HTLV I/II
- Active infection at the time of screening
- Diagnosis of Myelodysplastic Syndrome confirmed by FISH or karyotype
- Unable to undergo apheresis procedure
- Known hypersensitivity to murine proteins or iron-dextran
- Severe chronic infection
- Diseases or conditions posing risk to participant or interfering with study results
- History of malignancy
- Previous treatment with gene therapy, allogeneic bone marrow, or cord blood transplantation
- Change in growth hormone treatment regimen within 2 years prior to treatment
- Participation in another clinical trial or use of experimental medications within 1 month prior to this study
- Pregnant or intending to become pregnant within 12 months
- Deemed unsuitable for participation by the Investigator for any reason
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Single treatment with 12 months of safety follow-up
Participants receive a single intravenous infusion of autologous CD34+ cells enriched with allogeneic placenta-derived mitochondria.
Trial Site Locations
Total: 1 location
1
Sheba Medical Center
Ramat Gan, Israel, Israel, 5266202
Actively Recruiting
Research Team
L
Lea Bensoussan, Msc
N
Natalie Yivgi Ohana, PhD
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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