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 Phase 2 Interventional Study
Mitochondrial Diseases Pearson Syndrome
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 – 18
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
6 (estimated)
Sponsor
Minovia Therapeutics Ltd. · Industry
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About This Trial
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. Mitoc…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 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 manifestatio…
Contacts

Lea Bensoussan, Msc

+ 972 586101291

lea@minoviatx.com

CONTACT

Natalie Yivgi Ohana, PhD

+972 54 5833727

natalie@minoviatx.com

CONTACT