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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 treatment's 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 therapy's 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Follistatin plasmid | Genetic | Follistatin-344 plasmid |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 | 3 months | |
| Change from Baseline in Follistatin Serum Concentration at 3 months | 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry | 3 months | |
| Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry | 3 months | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Glenn Terry, MD | Principal Investigator | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Global Alliance for Regenerative Medicine | Roatán | Honduras |
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| ID | Term |
|---|---|
| D000073496 | Frailty |
| ID | Term |
|---|---|
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| Change in intrinsic and extrinsic epigenetic clock age |
| 3 months |