Status:
RECRUITING
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)
Lead Sponsor:
Minovia Therapeutics Ltd.
Conditions:
Mitochondrial Diseases
Pearson Syndrome
Eligibility:
All Genders
1-18 years
Phase:
PHASE2
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 dele...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
July 31 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT06017869
Start Date
July 31 2023
End Date
December 1 2027
Last Update
June 22 2025
Active Locations (1)
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1
Sheba Medical Center
Ramat Gan, Israel, Israel, 5266202