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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)

Offene Rekrutierung
Die Details der klinischen Studie sind hauptsächlich auf Englisch verfügbar. Trial Radar KI kann jedoch helfen! Klicken Sie einfach auf 'Studie erklären', um die Informationen zur Studie in der ausgewählten Sprache anzuzeigen und zu besprechen.
Die klinische Studie NCT06017869 untersucht Behandlung im Zusammenhang mit Mitochondriale Erkrankungen, Pearson-Syndrom. Diese interventionsstudie der Phase 2 hat den Status offene rekrutierung und startete am 31. Juli 2023. Es ist geplant, 6 Teilnehmer aufzunehmen. Durchgeführt von Minovia Therapeutics Ltd. wird der Abschluss für 1. Dezember 2027 erwartet. Die Daten von ClinicalTrials.gov wurden zuletzt am 22. Juni 2025 aktualisiert.
Kurzbeschreibung
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.

Offizieller Titel

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

Erkrankungen
Mitochondriale ErkrankungenPearson-Syndrom
Weitere Studien-IDs
  • MNV-010
NCT-Nummer
NCT06017869
Studienbeginn (tatsächlich)
2023-07-31
Zuletzt aktualisiert
2025-06-22
Studienende (vorauss.)
2027-12
Geplante Rekrutierung
6
Studientyp
Interventionsstudie
PHASE
Phase 2
Status
Offene Rekrutierung
Stichwörter
Autologous
Mitochondrial
Pearson
Transplantation
Stem cell
Primäres Ziel
Behandlung
Zuteilungsmethode
Nicht zutreffend
Interventionsmodell
Einarmige Studie
Verblindung
Keine (offene Studie)
Studienarme/Interventionen
Teilnehmergruppe/StudienarmIntervention/Behandlung
ExperimentellAutologous 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.
Hauptergebnismessungen
ErgebnismessungBeschreibung der MessungZeitrahmen
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
Nebenergebnismessungen
ErgebnismessungBeschreibung der MessungZeitrahmen
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
Eignungskriterien

Zugelassene Altersgruppen
Kind, Erwachsene
Mindestalter
1 Year
Zugelassene Geschlechter
Alle
  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.
Zentrale Studienkontakte
Kontakt: Lea Bensoussan, Msc, + 972 586101291, [email protected]
Kontakt: Natalie Yivgi Ohana, PhD, +972 54 5833727, [email protected]
1 Studienstandorte in 1 Ländern

Israel

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