Key Insights

Highlights

Success Rate

100% trial completion (above average)

Treatment Research

3 treatments under study, led by Ephedrine

Clinical Risk Assessment

Based on trial outcomes

Low Risk

Score: 15/100

Termination Rate

0.0%

0 terminated out of 4 trials

Success Rate

100.0%

+13.4% vs benchmark

Late-Stage Pipeline

25%

1 trials in Phase 3/4

Results Transparency

100%

1 of 1 completed with results

Key Signals

1 with results100% success

Data Visualizations

Phase Distribution

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

Trial Status

Unknown2
Completed1
Recruiting1

Trial Success Rate

100.0%

Benchmark: 86.6%

Based on 1 completed trials

Clinical Trials (4)

Showing 4 of 4 trials
NCT06630650RecruitingPrimary

A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes

NCT01765140UnknownPrimary

Treatment Use of 3,4-Diaminopyridine

NCT02562066Phase 3CompletedPrimary

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

NCT00541216Phase 1/2UnknownPrimary

Ephedrine for the Treatment of Congenital Myasthenia

Showing all 4 trials

Research Network

Common Treatments

  • Ephedrine1
  • Amifampridine Phosphate1
  • Amifampridine1

Active Sponsors

  • Hadassah Medical Organization1
  • Vern C. Juel, M.D.1
  • National Institute of Neurological Disorders and Stroke (NINDS)1
  • Catalyst Pharmaceuticals, Inc.1

Top Investigators

Activity Timeline