NCT04110964

Evaluation of Safety and Tolerability of Libella Gene Therapy for Critical Limb Ischemia: AAV- hTERT

Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Critical Limb Ischemia: AAV- hTERT

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Libella Gene Therapeutics · Industry
• Sex: All
• Age: 45 Years
• Healthy volunteers: Not accepted

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. 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 1\. Investigate the safety and tolerability of AAV-hTERT by intravenous (IV) administration. Secondary: Provisional Efficacy 1. Investigate LGT's ability to deliver hTERT to human cells and lengthen telomeres. 2. Investigate the effects of lengthening telomeres on CLI. 3. Investigate other benefits provided by lengthening telomeres.

Conditions

• Critical Limb Ischemia

Interventions

Type	Name	Description
DRUG	AAV-hTERT	subjects will receive a single LGT (AAV-hTERT) treatment via IV administration

Timeline

• Start date: 2019-09-26
• Primary completion: 2020-12-01
• Completion: 2020-12-01
• First posted: 2019-10-01
• Last updated: 2019-10-28

Locations

1 site across 1 country: Colombia