Key Insights

Highlights

Treatment Research

2 treatments under study, led by Gemfibrozil

Related Conditions

Often studied with 6 related conditions

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 50/100

Termination Rate

0.0%

0 terminated out of 2 trials

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed with results

Key Signals

Data Visualizations

Phase Distribution

1Total
Phase 2 (1)

Trial Status

Not Yet Recruiting1
Enrolling By Invitation1

Clinical Trials (2)

Showing 2 of 2 trials
NCT07740512Phase 2Not Yet Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

NCT03862274Enrolling By Invitation

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Showing all 2 trials

Research Network

Common Treatments

  • Gemfibrozil1
  • Cln2 Treatment1

Active Sponsors

  • Polaryx Therapeutics, Inc.1
  • BioMarin Pharmaceutical1
  • Jessica Scherr1

Top Investigators

Related Conditions

  • Sandhoff Disease1
  • Cln31
  • Neuronal Ceroidlipofuscinoses1
  • Lysosomal Storage Disorders1
  • Krabbe Disease1

Activity Timeline