Key Insights

Highlights

Treatment Research

4 treatments under study, led by Low Dose Cln301

Related Conditions

Often studied with 5 related conditions

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 30/100

Termination Rate

0.0%

0 terminated out of 3 trials

Late-Stage Pipeline

33%

1 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed with results

Key Signals

Data Visualizations

Phase Distribution

3Total
Phase 1/2 (1)
Phase 2 (1)
Phase 3 (1)

Trial Status

Not Yet Recruiting2
Active Not Recruiting1

Clinical Trials (3)

Showing 3 of 3 trials
NCT07882290Phase 3Not Yet RecruitingPrimary

Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease

NCT07740512Phase 2Not Yet Recruiting

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

NCT03770572Phase 1/2Active Not RecruitingPrimary

Gene Therapy for Children With CLN3 Batten Disease

Showing all 3 trials

Research Network

Common Treatments

  • Low Dose Cln3011
  • High Dose Cln3011
  • Gemfibrozil1
  • Miglustat Oral Liquid Formulation 250 Mgml1

Active Sponsors

  • Theranexus1
  • Polaryx Therapeutics, Inc.1
  • Beyond Batten Disease Foundation1
  • Neela Therapeutics1

Top Investigators

Related Conditions

  • Cln21
  • Krabbe Disease1
  • Lysosomal Storage Disorders1
  • Batten Disease1
  • Sandhoff Disease1

Activity Timeline