Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Gemfibrozil
PLX-200 · 1 trial · 1 indication
The change from baseline in the motor score of the Hamburg Rating Scale at Week 60 of maintenance therapy in participants treated with PLX-200 compared with that of Placebo group. Baseline is defined as the motor score of the Hamburg Rating Scale assessment prior to starting drug or Placebo group in the Titration Period. The Hamburg Rating Scale records a rating of 0 - 3 in four domains: motor skills, vision, language, and seizures, all from 0 - 3 which is worse to normal, respectively, with a minimum (worse) score of 0 to a maximum (normal) score of 12.
The aggregate of clinical chemistries, hematology, urinalysis, electrocardiogram readings, abdominal ultrasound findings, and tabulation of the number and severity of adverse events will be compared with baseline values throughout the study to evaluate the safety and tolerability of PLX-200 at escalating oral doses in children with CLN3 disease.
| Arm | Type | Description |
|---|---|---|
| PLX-200 | EXPERIMENTAL | This is randomized, placebo-controlled comparator study of PLX-200 in patients with CLN3 disease. |
| Placebo | PLACEBO_COMPARATOR | This is randomized comparator study of PLX-200 vs. placebo in a 2:1 ratio in patients with CLN3 disease. |
| Name | Type | Description |
|---|---|---|
| PLX-200 | DRUG | 15 mg/mL oral solution of experimental drug |
| Placebo | DRUG | Taste and color-matched drug-free solution |
Inclusion Criteria: 1. Male and female participants between the ages of 6 and 18 years of age. Any deviations from this age range must be approved by the Medical Monitor and Sponsor prior to entry into study. 2. Has a diagnosis of "classic" CLN3 disease as determined by age of symptom onset (i.e., ...
PLX-200 is being developed for juvenile neuronal ceroid lipofuscinosis, a rare inherited neurodegenerative disorder also known as CLN3 disease. It is a small molecule therapeutic candidate in the rare disease area. Polaryx Therapeutics is developing PLX-200 as an investigational treatment for patients with this condition.
PLX-200 is being developed by Polaryx Therapeutics, Inc., which trades under the ticker PLYX. The company is advancing the small molecule candidate for juvenile neuronal ceroid lipofuscinosis, a rare pediatric neurodegenerative disorder, as part of its rare disease pipeline.
PLX-200 is listed at Phase 3 for juvenile neuronal ceroid lipofuscinosis. It is an investigational small molecule and is not described as approved. Its clinical program consists of a single Phase 3 trial, NCT04637282, which was withdrawn with no patients enrolled.
PLX-200 has one registered clinical trial, NCT04637282, titled Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3. It was designed as a Phase 3 study in juvenile neuronal ceroid lipofuscinosis for patients aged 6 years and older, but the trial was withdrawn and enrolled no participants.
The PLX-200 trial NCT04637282 was a Phase 3 study in juvenile neuronal ceroid lipofuscinosis open to patients aged 6 years and older of any sex. It was an uncontrolled study without randomization or double blinding, and it was withdrawn before any of its planned enrollment took place.