Ignite Creation Date:
2024-05-19 @ 5:34 PM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06411366
Status:
COMPLETED
Last Update Posted:
2024-05-13
First Post:
2024-05-08
Brief Title:
Phase I Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Humans
Sponsor:
Minicircle
Organization:
Minicircle
Study Overview
Official Title:
A Phase I Open Label Single Dose Study to Evaluate the Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Healthy Subjects
Status:
COMPLETED
Status Verified Date:
2024-05
Last Known Status:
None
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:
This study examines the safety and effects of an injectable plasmid gene therapy Plasmids are circular pieces of DNA which have been widely studied as a non-permanent non-heritable method for transferring genes and inducing gene expression In this study the plasmid is a gene vector which contains the human FST344 gene intended to express and secrete bioidentical human follistatin into serum circulation Follistatin is a myostatin and activin inhibitor that has demonstrated improved functional outcomes in mouse models of neuromuscular disease
Participants will undergo dual energy x-ray absorptiometry scans before and after the treatments administration to compare change from baseline and rates of change in fat vs muscle tissue and bone density Participants will also undergo metabolic and epigenetic blood panels to observe any changes Participants will be monitored at the clinic site for a short time period after receiving the therapy and participants will be able to report any adverse events through an online form Lastly participants will have blood drawn just prior to and three months after the gene therapys administration in order to assess circulating levels of follistatin This study is administered at the Global Alliance for Regenerative Medicine clinical research site on the island of Roatan and is sponsored by Minicircle The main contact for this study is Mac Davis
Participants will undergo dual energy x-ray absorptiometry scans before and after the treatments administration to compare change from baseline and rates of change in fat vs muscle tissue and bone density Participants will also undergo metabolic and epigenetic blood panels to observe any changes Participants will be monitored at the clinic site for a short time period after receiving the therapy and participants will be able to report any adverse events through an online form Lastly participants will have blood drawn just prior to and three months after the gene therapys administration in order to assess circulating levels of follistatin This study is administered at the Global Alliance for Regenerative Medicine clinical research site on the island of Roatan and is sponsored by Minicircle The main contact for this study is Mac Davis
Detailed Description:
None
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