Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Phase 2RecruitingNCT07721025

Lexeo Therapeutics26 enrolled

Overview

The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Friedreich Ataxia Cardiomyopathy, Secondary

Interventions

LX2006GENETIC

Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Usual CareOTHER

Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover).

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable). * Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene) * Onset of FA on or before 25 years of age * Confirmed left ventricular hypertrophy and abnormal left ventricular mass index * Left ventricular ejection fraction at least 30% * Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level Exclusion Criteria: * Presence of other forms of cardiomyopathy that contribute to heart failure * Current use of inotrope infusion or presence of a ventricular assist device * Contraindication to cardiac MRI * Prior organ transplant * Previous gene transfer or cell therapy * Poorly controlled diabetes (hemoglobin A1c ≥8%) * Active hematologic or solid organ cancer Other inclusion/exclusion criteria to be applied as per protocol.

Locations (1)

University of South Florida

Tampa, Florida, United States

RECRUITING

Outcomes

Primary Outcomes

Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI

Time frame: At Week 26

Cohort 2: Incidence and severity of treatment-emergent adverse events

Time frame: Through Month 60

Cohort 2: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges

Time frame: Through Month 60

Cohort 2: Change in clinical vital signs (body temperature [°C])

Time frame: Through Month 60

Cohort 2: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])

Time frame: Through Month 60

Cohort 2: Change in clinical vital signs (heart rate [beats per minute])

Time frame: Through Month 60

Cohort 2: Change in clinical vital signs (respiratory rate [breaths per minute])

Time frame: Through Month 60

Cohort 2: Change in clinical vital signs (oxygen saturation [%])

Time frame: Through Month 60

Cohort 2: Change in clinical 12-lead ECG findings (ECG machine automatically calculates the heart rate [beats per minute] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex [milliseconds])

Time frame: Through Month 60

Secondary Outcomes

Cohorts 1 and 2: Change from baseline in high-sensitivity troponin I

Central Contacts

Lexeo Clinical Trials

CONTACT

212-547-9879 clinicaltrials@lexeotx.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Time frame: At Week 26

Cohort 1: Change from baseline in maximal wall thickness by cardiac MRI

Time frame: At Week 26

Cohort 1: Number of the following cardiovascular events as collected from the participant by the study doctor during scheduled study visits

* Death due to any cause * Hospitalization for heart failure * Non-fatal stroke * Non-fatal heart attack * Non-fatal life-threatening rapid or irregular heartbeat * Heart transplant * Insertion of a left ventricular assist device (pump) in the heart

Time frame: At Month 60

Cohort 1: Change from baseline in modified Friedreich's Ataxia Rating Scale

A neurological assessment tool for Friedreich ataxia that is measured on a scale of 0 to 93 with high scores indicating greater physical impairment

Time frame: At Week 26, at Week 52, and through Month 60

Cohort 1: Change from baseline in Kansas City Cardiomyopathy Questionnaire

A cardiac assessment tool scored from 0 to 100, with higher scores indicating better health status and lower scores reflecting more severe heart failure symptoms and functional limitations

Time frame: At Week 26, at Week 52, and through Month 60

Cohort 1: Incidence and severity of treatment-emergent adverse events

Time frame: Through Month 60

Cohort 1: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges

Time frame: Through Month 60

Cohort 1: Change in clinical vital signs (body temperature [°C])

Time frame: Through Month 60

Cohort 1: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])

Time frame: Through Month 60

Cohort 1: Change in clinical vital signs (heart rate [beats per minute])

Time frame: Through Month 60

Cohort 1: Change in clinical vital signs (respiratory rate [breaths per minute])

Time frame: Through Month 60

Cohort 1: Change in clinical vital signs (oxygen saturation [%])

Time frame: Through Month 60

Cohort 1: Change in clinical 12-lead ECG findings

ECG machine automatically calculates the heart rate \[beats per minute\] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex \[milliseconds\]

Time frame: Through Month 60

Cohort 2: Percent change from baseline in left ventricular mass index by cardiac MRI (or by ECHO for participants aged ≥6 to <12 years who did not have the cardiac MRI at baseline)

Time frame: At Week 26

Cohort 2: Change from baseline in maximal wall thickness by cardiac MRI (or by ECHO for participants aged ≥6 to <12 years who did not have the cardiac MRI at baseline)

Time frame: At Week 26