SUNRISE-FA 2 is a late-stage, clinical trial to evaluate whether the investigational gene therapy, LX2006, may improve heart function and limit the progression of Friedreich ataxia cardiomyopathy (FA-CM).
This is research that could bring us closer to addressing the root cause of FA-CM and change what is possible for the FA community. Your participation is key in getting us closer to answers.
The early-stage SUNRISE-FA trial showed improvements in heart structure and function. Participants treated with LX2006 experienced sustained reductions in left ventricular mass index (LVMi), a measure of heart thickening that is associated with serious cardiac complications in FA.1
Most participants also showed either improved or stable secondary cardiac biomarkers (additional measurements that show how well the heart is functioning) over time.
Participants currently enrolled in the CLARITY-FA natural history study may be eligible for participation in SUNRISE-FA 2 (eligibility to be determined by clinical trial doctor).
The information on this site is intended for U.S. residents only.
Overview*
Condition
The SUNRISE-FA 2 clinical trial is open to individuals with a confirmed genetic diagnosis of FA with evidence of cardiomyopathy based on specific criteria.
Population
The SUNRISE-FA 2 clinical trial will enroll individuals aged 16 years and older, followed by children aged 6 to under 16 years of age.
Administration
If you participate in this clinical trial, you will receive a one-time intravenous (IV) infusion of LX2006 as long as you remain eligible.
More details on the trial process can be found on the SUNRISE-FA 2 clinical trial timeline below.
*Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial.
SUNRISE-FA 2 Trial Timeline
Slide 1 of 7
SUNRISE-FA 2 Trial Overview
LX2006 is an investigational gene therapy* designed to deliver a working copy of the FXN gene to heart cells of individuals with FA-CM.
The goal: help your heart cells produce more of the frataxin protein they need and potentially improve heart function.
Slide 2 of 7
ELIGIBILITY SCREENING
Diagnosed individuals first take part in a thorough screening process to assess their eligibility.
Slide 3 of 7
RANDOMIZATION
If patients are eligible, they are then randomized into one of two pathways.
Only participants 16 years and older will be randomized. Children aged 6 to under 16 years of age will not be randomized and will move directly to IV infusion of LX2006 if eligible.
Slide 4 of 7
TRIAL PATHWAYS
Participants aged 16 years and older who are randomized to this pathway will undergo a 26-week untreated observation period. Following this observation phase, they will receive the investigational gene therapy, LX2006, delivered as a one-time intravenous (IV) infusion.
Slide 5 of 7
TRIAL PATHWAYS
Participants in this pathway will receive the investigational therapy, LX2006, delivered as a one-time intravenous (IV) infusion directly following randomization.
Slide 6 of 7
ASSESSMENTS & MONITORING
Following IV infusion of LX2006, all participants will be evaluated for 26 weeks.
Slide 7 of 7
LONG-TERM FOLLOW-UP FOR ALL PARTICIPANTS
Regardless of which pathway participants are randomized to, all participants will enter a long-term follow-up phase for an additional 4.5 years following the 26-week monitoring period.
Further details of the trial, including study visits, can be discussed with the Lexeo Clinical Trial Navigator.
*LX2006 is an investigational gene therapy meaning the safety and efficacy of LX2006 have not been established. LX2006 has not been approved by the U.S. Food and Drug Administration or any other country’s health authority or regulatory agency.
**SUNRISE-FA 2 is a randomized, open-label trial, which means randomization is used to determine which group each participant is in, but all participants will receive the investigational gene therapy, LX2006, regardless of pathway as long as they remain eligible.
Please note, only participants 16 years and older will be randomized. Children aged 6 to under 16 years of age will not be randomized and will move directly to IV infusion of LX2006 if eligible.
(IV = intravenous)