Ignite Creation Date:
2024-05-06 @ 3:10 PM
Last Modification Date:
2024-10-26 @ 1:44 PM
Study NCT ID:
NCT04548843
Status:
UNKNOWN
Last Update Posted:
2021-08-31
First Post:
2020-09-08
Brief Title:
A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC Autologous CD34 Cells Enriched With Placenta Derived Allogeneic Mitochondria in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
Sponsor:
Minovia Therapeutics Ltd
Organization:
Minovia Therapeutics Ltd
Study Overview
Official Title:
A First in Human Phase I Open Label Dose-escalation Study to Evaluate the Safety of Infusion of MNV-BM-PLC Autologous CD34 Cells Enriched With Placenta Derived Allogeneic Mitochondria in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
Status:
UNKNOWN
Status Verified Date:
2021-08
Last Known Status:
NOT_YET_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The study objectives are to evaluate the safety of a single intravenous IV infusion of autologous CD34 cells enriched with placenta-derived allogeneic mitochondria in participant with primary mitochondrial disease associated with mitochondrial DNA mutations or deletions
6 participants aged from 4 to 18 years old on the day of screening visit with primary mitochondrial disease associated with mitochondrial DNA mutations or deletions will be enrolled
6 participants aged from 4 to 18 years old on the day of screening visit with primary mitochondrial disease associated with mitochondrial DNA mutations or deletions will be enrolled
Detailed Description:
MNV-BM-PLC is a personalized cell therapy based on autologous patient-derived Hematopoietic stemprogenitor cells HSPCs enriched with mitochondria isolated from healthy placenta obtained from donors during C-section Healthy mitochondria are employed ex-vivo to enrich the patients CD34 peripheral blood cells followed by infusion of the mitochondrial enriched cells back to the patient This therapeutic process of mitochondrial augmentation provides the patient with healthy mitochondria carrying non-mutateddeleted mtDNA that can supplement mitochondrial functionality in the patients cells
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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