Critical Limb IschemiaPhase 1NCT04110964
Evaluation of Safety and Tolerability of Libella Gene Therapy for Critical Limb Ischemia: AAV- hTERT
A Phase 1 study testing AAV-hTERT in people with Critical Limb Ischemia, run by Libella Gene Therapeutics at 1 site in Colombia.
Trial summary
Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many diseases related to aging, including critical limb ischemia (CLI). This study will entail treating subjects with hTERT delivered via transduction using AAV. Read more about this study
- Who is it for?
- Ages 45 years and older
- Conditions the study is for
- Critical Limb Ischemia
- What is being tested?
- AAV-hTERT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteColombia
- Study dates
- 26 Sep 2019to Dec 2020
- 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 45 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age of 45 years or older.
- Clinical diagnosis of CLI.
- Subjects, or their authorized representatives, must be adequately informed and understand the nature and risks of the study and must be able to provide a signature and date in the Informed Consent Form (ICF).
- Women must undergo a negative serum pregnancy test at the screening assessment.
- Subjects, or their representatives, must be able to communicate effectively with the study staff.
- Subjects, or their authorized representatives, must certify that they are able and willing to follow all protocol requirements and study restrictions.
Who cannot join
- Subjects who have a history of allergy, hypersensitivity, or intolerance to any medications, components, or excipients of the investigational product, which cannot be resolved by the staff conducting the study.
- Female subjects who are pregnant, expected to become pregnant, or lactating/nursing.
- Subjects who are sexually active and who are unwilling or unable to use a method of effective contraception (e.g., hormonal and/or barrier) during their participation in the study.
- Subjects who are intolerant of, or do not wish to receive IV injections.
- Subjects who are currently alcoholics and/or use psychoactive substances.
- Subjects who cannot tolerate venipuncture and/or venous access.
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- Subjects who have donated or had a significant loss of whole blood (480 ml or more) within 30 days, or donated plasma or platelets within 14 days prior to screening.
- Subjects who have received blood or blood products within 30 days prior to screening.
- Subjects who have been treated with another research product 30 days prior to the screening assessment, or plan to participate in another clinical trial, while in this study if in the opinion of the principal investigator, may place the subject at risk due to participation in the study, or may influence the results of the study or the subject's ability to complete the study. If more than 30 days have passed since participation in another clinical trial, the study staff must ensure that the subject has recovered from any adverse event (AE) associated with the research product used.
- Subjects who have a history or evidence of active infection or febrile illness within 7 days prior to the screening assessment.
- Subjects who have a history of any other clinically significant disease or disorder that, in the opinion of the principal investigator, may place the subject at risk due to participation in the study, or may influence the results of the study or the subject's ability to complete the study.
- -
Trial locations and contacts
1 site in 1 country.
Colombia1 site, 1 recruiting
- Recruiting
IPS Arcasalud SAS
Zipaquirá, Cundinamarca, 250251
Wiston Pernet, MD, 57 3006840951, wirapeca@gmail.com
Jorge Ulloa, MD
About this study
Official title: Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Critical Limb Ischemia: AAV- hTERT
Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many diseases related to aging, including critical limb ischemia (CLI).
This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of CLI. It is expected to have a direct consequence on function and quality of life in patients with Peripheral artery disease (PAD); in this case a subgroup of patients with CLI, the worst presentation of PAD.
Read the detailed description
Patients diagnosed with CLI who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravascularly (IV).
Baseline will be performed within 8 weeks of beginning the treatment regimen. The treatment regimen will begin with IV delivery of AAV-hTERT, defined as "Day 0." Safety and efficacy analyses will be conducted at Weeks 1, 4, 13, 26, 39, and 52 post-treatment.
Study objectives
Primary: Safety and Tolerability
- Investigate the safety and tolerability of AAV-hTERT by intravenous (IV) administration.
Secondary: Provisional Efficacy
- Investigate LGT's ability to deliver hTERT to human cells and lengthen telomeres.
- Investigate the effects of lengthening telomeres on CLI.
- Investigate other benefits provided by lengthening telomeres.
- Conditions
- Critical Limb Ischemia
- Being tested
- AAV-hTERT
- Keywords
- Telomeres, Telomerase, Critical limb ischemia, PAD, AAV, Gene therapy
- Medical subject headings
- Chronic Limb-Threatening Ischemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV-hTERTDrug
Also called LGT
subjects will receive a single LGT (AAV-hTERT) treatment via IV administration
| Study group | Role | What they receive |
|---|---|---|
| subjects treated with LGT | Experimental | Subjects will be treated with a single IV dose of LGT (AAV-hTERT) Receives: Drug: AAV-hTERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Incidence of serious adverse events and adverse events throughout the study
Measured: 12 months
Secondary outcomes (1)
hTERT expression and telomerase activity
Change from baseline of the telomere length
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2019
- Study start26 Sep 2019
- Primary completionDec 2020 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Critical Limb IschemiaStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Libella Gene Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jorge Ulloa, MD, Principal investigator, IPS Arcaslud SAS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04110964
- Sponsor's protocol ID
- LibellaCO-01
- Status last verified
- Oct 2019
- First submitted
- 27 Sep 2019
- Last updated
- 28 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Critical Limb Ischemia: AAV- hTERT
- ClinicalTrials.gov ID
- NCT04110964
- Acronym / sponsor ID
- LibellaCO-01
- Conditions
- Critical Limb Ischemia
- Keywords
- Telomeres, Telomerase, Critical limb ischemia, PAD, AAV, Gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Libella Gene TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 26 Sep 2019 (actual)
- Primary completion
- Dec 2020 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 1 Oct 2019 (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.
Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many diseases related to aging, including critical limb ischemia (CLI).
This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of CLI. It is expected to have a direct consequence on function and quality of life in patients with Peripheral artery disease (PAD); in this case a subgroup of patients with CLI, the worst presentation of PAD.
Detailed description
Patients diagnosed with CLI who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravascularly (IV).
Baseline will be performed within 8 weeks of beginning the treatment regimen. The treatment regimen will begin with IV delivery of AAV-hTERT, defined as "Day 0." Safety and efficacy analyses will be conducted at Weeks 1, 4, 13, 26, 39, and 52 post-treatment.
Study objectives
Primary: Safety and Tolerability
- Investigate the safety and tolerability of AAV-hTERT by intravenous (IV) administration.
Secondary: Provisional Efficacy
- Investigate LGT's ability to deliver hTERT to human cells and lengthen telomeres.
- Investigate the effects of lengthening telomeres on CLI.
- Investigate other benefits provided by lengthening telomeres.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| subjects treated with LGT | Experimental | Drug: AAV-hTERTSubjects will be treated with a single IV dose of LGT (AAV-hTERT) |
Who may qualify
- Ages
- 45 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age of 45 years or older.
- Clinical diagnosis of CLI.
- Subjects, or their authorized representatives, must be adequately informed and understand the nature and risks of the study and must be able to provide a signature and date in the Informed Consent Form (ICF).
- Women must undergo a negative serum pregnancy test at the screening assessment.
- Subjects, or their representatives, must be able to communicate effectively with the study staff.
Key exclusion criteria (12 listed)
- Subjects who have a history of allergy, hypersensitivity, or intolerance to any medications, components, or excipients of the investigational product, which cannot be resolved by the staff conducting the study.
- Female subjects who are pregnant, expected to become pregnant, or lactating/nursing.
- Subjects who are sexually active and who are unwilling or unable to use a method of effective contraception (e.g., hormonal and/or barrier) during their participation in the study.
- Subjects who are intolerant of, or do not wish to receive IV injections.
- Subjects who are currently alcoholics and/or use psychoactive substances.
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 |
|---|---|---|
| AAV-hTERTAlso: LGTsubjects will receive a single LGT (AAV-hTERT) treatment via IV administration | Drug | subjects treated with LGT |
Primary outcome
Incidence of adverse events
Time frame: 12 months
Incidence of serious adverse events and adverse events throughout the study
1 secondary outcome
hTERT expression and telomerase activity
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2019.
- Individual participant dataSharing planned: No
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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.
Jorge Ulloa, MD principal investigator
IPS Arcaslud SAS
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 AAV-hTERT in people with Critical Limb Ischemia, run by Libella Gene Therapeutics at 1 site in Colombia.
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 6 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 28 Oct 2019. 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 Colombia. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jeff Mathis, PhD (1 785 4100223, jmathis@libellagt.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: 12 months). 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 28 Oct 2019.