Key Insights

Highlights

Success Rate

100% trial completion (above average)

Research Leaders

20 sponsors active, led by Cure Mito Foundation

Related Conditions

Often studied with 20 related conditions

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 50/100

Termination Rate

0.0%

0 terminated out of 2 trials

Success Rate

100.0%

+13.4% vs benchmark

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 1 completed with results

Key Signals

100% success

Data Visualizations

Phase Distribution

Trial Status

Completed1
Recruiting1

Trial Success Rate

100.0%

Benchmark: 86.6%

Based on 1 completed trials

Clinical Trials (2)

Showing 2 of 2 trials
NCT01793168Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

NCT02381457Completed

SNP-based Microdeletion and Aneuploidy RegisTry (SMART)

Showing all 2 trials

Research Network

Active Sponsors

  • Cure Mito Foundation1
  • PROS Foundation1
  • SHINE-Syndrome Foundaion1
  • Acrodysostosis Support and Research1
  • Cornelia de Lange Syndrome Foundation1

Top Investigators

Related Conditions

  • Cerebellar Ataxia With Peripheral Neuropathy1
  • Adult Hypophosphatasia1
  • Betamannosidosis1
  • Idiopathic Gastroparesis1
  • Cerebellar Ataxia Areflexia Pes Cavus Optic Atrophy Sensorineural Hearing Loss1

Activity Timeline