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Recruiting NCT07721025

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Phase II Interventional Friedreich Ataxia Cardiomyopathy, Secondary

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: LX2006, Usual Care.
Who it may be relevant to
Registry conditions: Friedreich Ataxia, Cardiomyopathy, Secondary. Basic parameters: from 6 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

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).

Interventions

  • Genetic LX2006
    Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
  • Other Usual Care
    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).

Primary outcome measures

  • Percent change from baseline in left ventricular mass index by cardiac MRI [Time frame: At Week 26]
Secondary outcome measures (12)
  • Change from baseline in high-sensitivity troponin I [Time frame: At Week 26 and through Month 60]
  • Change from baseline in left ventricular wall thickness and additional imaging outcomes [Time frame: At Week 26 and through Month 60]
  • Change from baseline in modified Friedreich's Ataxia Rating Scale [Time frame: At Week 26 and through Month 60]
  • Change from baseline in Kansas City Cardiomyopathy Questionnaire [Time frame: Through Month 60]
  • Healthcare resource use [Time frame: Through Month 60]
  • Cardiovascular events [Time frame: Through Month 60]
  • Patient Global Impressions scales [Time frame: Through Month 60]
  • Incidence and severity of treatment-emergent adverse events [Time frame: Through Month 60]
  • Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges [Time frame: Through Month 60]
  • Change in clinical vital signs (body temperature [°C]) [Time frame: Through Month 60]
  • Change in clinical vital signs (systolic and diastolic blood pressure [mmHg]) [Time frame: Through Month 60]
  • Change in clinical vital signs (heart rate [beats per minute]) [Time frame: Through Month 60]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Crossover
Masking
Single blind
Primary purpose
Treatment

Study locations

United States · 1 center
  • University of South Florida — Tampa

Identifiers

NCT: NCT07721025 · LX2006-03 · 2025-524970-40-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗