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

NCT04110964 | Unknown Phase 1 DMC

• 1 other identifier: LibellaCO-01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Critical Limb Ischemia

Keywords

Telomeres; Telomerase; Critical limb ischemia; PAD; AAV; Gene therapy

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  Incidence of serious adverse events and adverse events throughout the study

  12 months

Secondary Outcomes (1)

• hTERT expression and telomerase activity

  12 months

Study Arms (1)

subjects treated with LGT

EXPERIMENTAL

Subjects will be treated with a single IV dose of LGT (AAV-hTERT)

Drug: AAV-hTERT

Interventions

AAV-hTERT DRUG

subjects will receive a single LGT (AAV-hTERT) treatment via IV administration

Also known as: LGT

subjects treated with LGT

Eligibility Criteria

• Age: 45 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Libella Gene Therapeutics: lead

Study Sites (1)

IPS Arcasalud SAS

Zipaquirá, Cundinamarca, 250251, Colombia

RECRUITING

MeSH Terms

Conditions

Chronic Limb-Threatening Ischemia

Condition Hierarchy (Ancestors)

Peripheral Arterial Disease; Atherosclerosis; Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases; Peripheral Vascular Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Ischemia

Study Officials

• Jorge Ulloa, MD

  IPS Arcaslud SAS

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jeff Mathis, PhD

CONTACT

1 785 4100223; jmathis@libellagt.com

Osvaldo Martinez-Clark

CONTACT

1 786 471-7814; ozclark@libellagt.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2019
• First Posted: October 1, 2019
• Study Start: September 26, 2019
• Primary Completion: December 1, 2020
• Study Completion: December 1, 2020
• Last Updated: October 28, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

CO (1)