Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 5 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 2 completed trials have results

Key Signals

1 recruiting

Enrollment Performance

Analytics

Phase 1
1(100.0%)
1Total
Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (5)

Showing 5 of 5 trials
NCT03897361Phase 1Completed

Stem Cell Gene Therapy for Cystinosis

Role: collaborator

NCT05545774Completed

Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients

Role: collaborator

NCT01793168Recruiting

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

Role: collaborator

NCT05146830Enrolling By Invitation

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Role: collaborator

NCT01327807Unknown

Cure Cystinosis International Registry

Role: lead

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