Polaryx Therapeutics is developing a patient-friendly oral small molecule therapy for Juvenile Neuronal Ceroid Lipofuscinosis, also known as CLN3 disease. It is marked by lysosomal dysfunction, resulting in lipofuscin accumulation throughout the body, seizures, vision loss, locomotor and cognitive dysfunctions, and eventually premature death.
No drug is currently available to halt or delay disease progression. The standard of care only manages symptoms. Therefore, Polaryx has explored relevant drug candidates, and identified PLX-200 to treat CLN3 disease. PLX-200 contains an FDA-approved lipid-lowering agent. Efficacy studies in a CLN3 animal disease model showed that PLX-200 reduces neuroinflammation, neuronal cell death, and accumulation of storage materials in the brain, thus delays the decline in motor function. As such, the efficacy of PLX-200 in the CLN3 disease model provides the scientific rationale to test with CLN3 patients.
Polaryx will perform a Randomized, Multicenter, Double-Blind, Placebo-Controlled Safety, Tolerability, and Efficacy Study in Participants with Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease. Polaryx named this trial the STARLIGHT trial.
In order to participate in the STARLIGHT trial, your child would need to meet pre-specified eligibility criteria.
At a minimum, these criteria include:
- 6 to 18 years of age.
- Participant must have a mild-to-moderate CLN3 disease and be able to independently walk at least 20 feet.
- Participant must be able to tolerate swallowing oral medication.
The STARLIGHT trial focuses on two key aspects, including the efficacy of PLX-200 and the safety of patients.
If you need more information about the STARLIGHT trial, please contact Dr. Hahn-Jun Lee, M.Sc., Ph.D. at email@example.com.
More information on the STARLIGHT trial can be found at Clinicaltrials.gov NCT04637282.