Health TrialsbyHealthPanel

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

ConditionsFriedreich AtaxiaCardiomyopathy, Secondary
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorLexeo Therapeutics

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Crossover

Treatments tested in this trial

  • LX2006

    Genetic

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

  • Usual Care

    Other intervention

    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).

Treatment groups

26 Participants
are divided into 2 treatment groups
Group A: LX2006Experimental treatment 1 intervention
Group B: Usual CareOther 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
At Week 26
2

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

Time frame
Through Month 60
3

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

Time frame
Through Month 60
4

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

Time frame
Through Month 60

Secondary outcomes

1

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

Time frame
At Week 26
2

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

Time frame
At Week 26
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Lexeo Therapeutics

Lead sponsor

Source ClinicalTrials.gov
Apply now