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Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3

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NCT04637282 · readout in 134 d

Sponsored by Polaryx Therapeutics, Inc. (industry) · PLYX — their whole pipeline →.

Phase
Phase 3
Status
Withdrawn
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
0actual

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Why the sponsor stopped it

Study has yet to begin and removing to avoid further participant outreach to sponsor

As filed on the registry record, in the sponsor's own words.

Dates

each axis at the precision it was filed, with the registry's own basis
DateFiledBasisWhat it is
Start2026-11-01estimatedWhen the study began enrolling
Primary completion2026-12-31estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completion2026-12-31estimatedThe whole study's end, after follow-up
First posted2020-11-19actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-08-19actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Juvenile Neuronal Ceroid Lipofuscinosis

Interventions

  • Drug: PLX-200 — also filed as Gemfibrozil
  • Drug: Placebo

Primary outcomes

what the primary-completion date above is the date OF
Efficacy of PLX-200 in CLN3 as assessed by the change in the motor score of the Hamburg Rating Scale compared with that of the Placebo group
measured 60 weeks
Number of patients with treatment-related adverse events, as assessed by CTCAE v5.0, abnormal laboratory results, and abnormal cardiovascular and/or abdominal findings.
measured 96 weeks

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