Clinical Trials Logo
  1. Home
  2. Pearson Syndrome
  3. NCT03384420

A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

Mitochondrial Diseases Clinical Trial

Official title:
A Phase I/II, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD (Autologous cd34+ Cells Enriched With Blood Derived Mitochondria) in Pediatric Patients With Pearson Syndrome

Clinical Trial Summary

Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes. These diseases may be inherited from the mother (mitochondrial inheritance) or non-inherited. The latter are ultra-rare pediatric diseases caused by a mutation or deletion of mtDNA, which develop into a systemic multi organ disease and eventually death. MNV-BM-BLD is a therapeutic process for enrichment of patient's peripheral hematopoietic stem cells with normal and healthy mitochondria derived from donor blood cells. The process, called mitochondria augmentation therapy, aims to reduce the symptoms of mitochondrial diseases.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03384420
Study type Interventional
Source Minovia Therapeutics Ltd.
Contact
Status Completed
Phase Phase 1/Phase 2
Start date February 13, 2019
Completion date March 9, 2021
View Details
See also
  Status Clinical Trial Phase
Completed NCT03388528 - Low Residue Diet Study in Mitochondrial Disease N/A
Completed NCT04378075 - A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy Phase 2/Phase 3
Completed NCT03678740 - Diagnostic Odyssey Survey 2
Recruiting NCT06051448 - Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD). Phase 1/Phase 2
Completed NCT02909400 - The KHENERGY Study Phase 2
Completed NCT02398201 - A Study of Bezafibrate in Mitochondrial Myopathy Phase 2
Completed NCT03857880 - Identification of New Candidate Genes in Patients With Mitochondrial Disease by High Resolution Chromosome Analysis on DNA Chip
Not yet recruiting NCT06450964 - Establishment of Reproductive Cohort and Prediction Model of Genetic Counseling for Mitochondrial Genetic Diseases
Completed NCT04165239 - The KHENERGYZE Study Phase 2
Completed NCT02284334 - Glycemic Index in Mitochondrial Disease
Recruiting NCT06080581 - Mitochondrial Dysfunctions Driving Insulin Resistance
Recruiting NCT06080568 - Human Mitochondrial Stress-driven Obesity Resistance
Recruiting NCT04802707 - Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome Phase 2
Completed NCT04580979 - Natural History Study of FDXR Mutation-related Mitochondriopathy
Completed NCT04594590 - Natural History Study of SLC25A46 Mutation-related Mitochondriopathy
Withdrawn NCT03866954 - Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy Phase 2
Recruiting NCT04113447 - Mitochondrial Donation: An 18 Month Outcome Study.
Enrolling by invitation NCT04734626 - CrCest Study in Primary Mitochondrial Disease
Completed NCT03832218 - Executive Function Disorders and Anxio-depressive Symptomatology in Children and Adolescents With Mitochondrial Pathologies N/A
Terminated NCT02473445 - A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease Phase 2