Mitochondrial Diseases Clinical Trial
Official title:
A Phase I, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
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.
n/a
| 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 |