Key Insights

Highlights

Success Rate

100% trial completion (above average)

Treatment Research

2 treatments under study, led by Hg204

Research Leaders

10 sponsors active, led by Boston Children's Hospital

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 trials in Phase 3/4

Results Transparency

0 of 1 completed with results

Key Signals

100% success

Data Visualizations

Phase Distribution

1Total

Not Applicable (1)

Trial Status

Recruiting1

Completed1

Trial Success Rate

100.0%

Benchmark: 86.6%

Based on 1 completed trials

Clinical Trials (2)

Showing 2 of 2 trials

NCT06615206Not ApplicableRecruitingPrimary

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

NCT03077308Completed

Rare Diseases Clinical Research Network: Neurophysiological Correlates

Showing all 2 trials

Research Network

Common Treatments

Hg2041
Auditory And Visual Eventrelated Potentials And Eeg1

Active Sponsors

Boston Children's Hospital1
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)1
Children's Hospital of Philadelphia1
Peking University First Hospital1
University of South Florida1

MECP2 Duplication Syndrome