FrailtyPhase 1NCT06411366
Phase I: Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Humans
A Phase 1 study testing Follistatin plasmid in people with Frailty and Aging, run by Minicircle at 1 site in Honduras.
Trial 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. Read more about this study
- Who is it for?
- Ages 22 years and olderHealthy volunteers can take part
- What is being tested?
- Follistatin plasmid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 1 siteHonduras
- Study dates
- 18 Aug 2022to 31 Aug 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 22 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Openness to morphological change
- Will practice contraception for the duration of trial (if female)
Who cannot join
- Women who are pregnant, nursing or of childbearing potential who are unwilling or unable to maintain contraceptive therapy for the duration of the study
- Life expectancy < 6 months due to concomitant illness
- Pre-existing joint, ligament, or heart issues
- Systolic blood pressure (supine) ≤90 mmHg;
- Resting heart rate > 100 bpm
- Unwilling or unable to give written informed consent.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Being tested
- Follistatin plasmid
- Medical subject headings
- Frailty
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Follistatin plasmidGenetic
Follistatin-344 plasmid
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | Not described Receives: Genetic: Follistatin plasmid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Measured: 3 months
Change from Baseline in Follistatin Serum Concentration at 3 months
Measured: 3 months
Secondary outcomes (3)
Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry
Measured: 3 months
Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry
Measured: 3 months
Change in intrinsic and extrinsic epigenetic clock age
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2024
- Study start18 Aug 2022
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FrailtyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Minicircle
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Glenn Terry, MD, Principal investigator, Principal Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06411366
- Sponsor's protocol ID
- FST-X7
- Status last verified
- May 2024
- First submitted
- 8 May 2024
- Last updated
- 13 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06411366
- Acronym / sponsor ID
- FST-X7
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- MinicircleIndustry (pharma, biotech, devices)
- Study start
- 18 Aug 2022 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 13 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Genetic: Follistatin plasmid |
Who may qualify
- Ages
- 22 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Openness to morphological change
- Will practice contraception for the duration of trial (if female)
Key exclusion criteria (6 listed)
- Women who are pregnant, nursing or of childbearing potential who are unwilling or unable to maintain contraceptive therapy for the duration of the study
- Life expectancy < 6 months due to concomitant illness
- Pre-existing joint, ligament, or heart issues
- Systolic blood pressure (supine) ≤90 mmHg;
- Resting heart rate > 100 bpm
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Follistatin plasmidFollistatin-344 plasmid | Genetic | Experimental |
Primary outcome
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Time frame: 3 months
Change from Baseline in Follistatin Serum Concentration at 3 months
Time frame: 3 months
3 secondary outcomes
Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry
Time frame: 3 months
Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry
Time frame: 3 months
Change in intrinsic and extrinsic epigenetic clock age
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Glenn Terry, MD principal investigator
Principal Investigator
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Follistatin plasmid in people with Frailty and Aging, run by Minicircle at 1 site in Honduras.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Honduras. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2024.