Phase
Condition
Mitochondrial Diseases
Treatment
MNV-201
Clinical Study ID
Ages 1-18 All Genders
Study Summary
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 molecularidentification of deletion in mtDNA of peripheral blood. Participants are diagnosedwith PS Participant can be in either the PS manifestations of the disease or mayhave transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history ofPS.
Participants have failure to thrive (height SDS smaller than -1)
Participants should have at least 12 months' history of body weight and height andcalculated GFR (from creatinine) before treatment.
Body weight ≥ 10 kg.
Participants' living parent(s) and/or legal guardian(s) able to understand andprovide voluntary written informed consent.
Participants' parents or legal guardian have a good understanding of the study andnature of the procedure and are expected to be able to comply with study visitschedules and caregiver assessments without difficulty.
Participants' parents or legal guardian provides written informed consent prior tostudy participation.
Participants are medically able to undergo the study interventions as determined bythe Investigator.
Exclusion
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/orkaryotype.
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 interferewith the ability to interpret the study results.
History of malignancy.
History of treatment with gene therapy, allogeneic bone marrow or cord bloodtransplantation.
Participants have had a change in growth hormone regimen in less than 2 years priorto treatment.
Participants have participated in another clinical trial or received otherexperimental medications outside a clinical trial within 1 month prior to start ofthis 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 participatingin the study for any reason.
Study Design
Connect with a study center
Sheba Medical Center
Ramat Gan, 5266202
IsraelActive - Recruiting

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