Evaluation of Safety and Tolerability of Libella Gene Therapy for Critical Limb Ischemia: AAV- hTERT
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|ClinicalTrials.gov Identifier: NCT04110964|
Recruitment Status : Recruiting
First Posted : October 1, 2019
Last Update Posted : October 28, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Critical Limb Ischemia||Drug: AAV-hTERT||Phase 1|
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.
Primary: Safety and Tolerability
1. 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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Critical Limb Ischemia: AAV- hTERT|
|Actual Study Start Date :||September 26, 2019|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||December 2020|
Experimental: subjects treated with LGT
Subjects will be treated with a single IV dose of LGT (AAV-hTERT)
subjects will receive a single LGT (AAV-hTERT) treatment via IV administration
Other Name: LGT
- Incidence of adverse events [ Time Frame: 12 months ]Incidence of serious adverse events and adverse events throughout the study
- hTERT expression and telomerase activity [ Time Frame: 12 months ]Change from baseline of the telomere length
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04110964
|Contact: Jeff Mathis, PhD||1 785 email@example.com|
|Contact: Osvaldo Martinez-Clark||1 786 firstname.lastname@example.org|
|IPS Arcasalud SAS||Recruiting|
|Zipaquirá, Cundinamarca, Colombia, 250251|
|Contact: Wiston Pernet, MD 57 3006840951 email@example.com|
|Principal Investigator: Jorge Ulloa, MD|
|Principal Investigator:||Jorge Ulloa, MD||IPS Arcaslud SAS|