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New treatment option for Friedreich Ataxia
Official title Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
ClinicalTrials.gov ID: NCT07721025
What this study is testing
What is LX2006?
LX2006 is an investigational medicine, being studied as a potential treatment for friedreich ataxia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- 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%
You likely can't join if
- 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%)
See the full eligibility 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
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Tampa, Florida, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tampa, Florida, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.