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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04110964
Recruitment Status : Recruiting
First Posted : October 1, 2019
Last Update Posted : October 28, 2019
Sponsor:
Information provided by (Responsible Party):
Libella Gene Therapeutics

Tracking Information
First Submitted Date  ICMJE September 27, 2019
First Posted Date  ICMJE October 1, 2019
Last Update Posted Date October 28, 2019
Actual Study Start Date  ICMJE September 26, 2019
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 28, 2019)
Incidence of adverse events [ Time Frame: 12 months ]
Incidence of serious adverse events and adverse events throughout the study
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT04110964 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 24, 2019)
hTERT expression and telomerase activity [ Time Frame: 12 months ]
Change from baseline of the telomere length
Original Secondary Outcome Measures  ICMJE
 (submitted: September 28, 2019)
  • hTERT expression and telomerase activity [ Time Frame: 12 months ]
    Change from baseline of the telomere length
  • A) CLI progression [ Time Frame: 12 months ]
    Change from baseline of the thermography results
  • B) CLI progression [ Time Frame: 12 months ]
    Change from baseline of the claudicometry
  • C) CLI progression [ Time Frame: 12 months ]
    Change from baseline of the Ankle Brachial Index
  • D) CLI progression [ Time Frame: 12 months ]
    Change from baseline of the Visual Analogue Scale for pain results
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluation of Safety and Tolerability of Libella Gene Therapy for Critical Limb Ischemia: AAV- hTERT
Official Title  ICMJE Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Critical Limb Ischemia: AAV- hTERT
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.

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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Critical Limb Ischemia
Intervention  ICMJE Drug: AAV-hTERT
subjects will receive a single LGT (AAV-hTERT) treatment via IV administration
Other Name: LGT
Study Arms  ICMJE Experimental: subjects treated with LGT
Subjects will be treated with a single IV dose of LGT (AAV-hTERT)
Intervention: Drug: AAV-hTERT
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 28, 2019)
5
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age of 45 years or older.
  2. Clinical diagnosis of CLI.
  3. 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).
  4. Women must undergo a negative serum pregnancy test at the screening assessment.
  5. Subjects, or their representatives, must be able to communicate effectively with the study staff.
  6. Subjects, or their authorized representatives, must certify that they are able and willing to follow all protocol requirements and study restrictions.

Exclusion Criteria:

  1. 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.
  2. Female subjects who are pregnant, expected to become pregnant, or lactating/nursing.
  3. 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.
  4. Subjects who are intolerant of, or do not wish to receive IV injections.
  5. Subjects who are currently alcoholics and/or use psychoactive substances.
  6. Subjects who cannot tolerate venipuncture and/or venous access.
  7. 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.
  8. Subjects who have received blood or blood products within 30 days prior to screening.
  9. 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.
  10. 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.

-

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 45 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jeff Mathis, PhD 1 785 4100223 jmathis@libellagt.com
Contact: Osvaldo Martinez-Clark 1 786 471-7814 ozclark@libellagt.com
Listed Location Countries  ICMJE Colombia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04110964
Other Study ID Numbers  ICMJE LibellaCO-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Libella Gene Therapeutics
Study Sponsor  ICMJE Libella Gene Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jorge Ulloa, MD IPS Arcaslud SAS
PRS Account Libella Gene Therapeutics
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP