Ignite Creation Date:
2024-05-06 @ 10:54 AM
Last Modification Date:
2024-10-26 @ 12:37 PM
Study NCT ID:
NCT03384420
Status:
COMPLETED
Last Update Posted:
2021-08-31
First Post:
2017-11-30
Brief Title:
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Sponsor:
Minovia Therapeutics Ltd
Organization:
Minovia Therapeutics Ltd
Study Overview
Official Title:
A Phase III 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
Status:
COMPLETED
Status Verified Date:
2020-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 patients 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None