Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

10.0%

2 terminated/withdrawn out of 20 trials

Success Rate

88.2%

+1.7% vs industry average

Late-Stage Pipeline

15%

3 trials in Phase 3/4

Results Transparency

47%

7 of 15 completed trials have results

Key Signals

1 recruiting7 with results

Enrollment Performance

Analytics

Phase 2

5(38.5%)

Phase 3

3(23.1%)

Phase 1

2(15.4%)

Early Phase 1

2(15.4%)

N/A

1(7.7%)

13Total

Phase 2(5)

Phase 3(3)

Phase 1(2)

Early Phase 1(2)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (20)

Showing 20 of 20 trials

NCT02255435Phase 2Completed

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

Role: collaborator

NCT06496451Completed

Measurement of Frataxin mRNA in Biofluids

Role: collaborator

NCT05168774Phase 1Completed

FRDA Investigator Initiated Study (IIS) With Elamipretide

Role: collaborator

NCT06016946Recruiting

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Role: lead

NCT06560346Withdrawn

Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

Role: lead

NCT03418740Completed

Neurology Measures in FA Children

Role: collaborator

NCT02593773Phase 3Completed

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study

Role: collaborator

NCT02415127Phase 3Completed

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

Role: collaborator

NCT02797080Phase 3Completed

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

Role: collaborator

NCT04349514Active Not Recruiting

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

Role: collaborator

NCT03090789Active Not Recruiting

FA Clinical Outcome Measures

Role: lead

NCT04649866Not ApplicableCompleted

Coronary Artery Disease in Patients With Friedreich's Ataxia

Role: collaborator

NCT01776164Completed

Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia

Role: collaborator

NCT01965327Phase 2Completed

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Role: collaborator

NCT02705547Early Phase 1Completed

Rosuvastatin (Crestor) in Friedreich Ataxia

Role: collaborator

NCT02424435Early Phase 1Completed

Methylprednisolone Treatment of Friedreich Ataxia

Role: collaborator

NCT01962363Phase 2Completed

EPI-743 in Friedreich's Ataxia Point Mutations

Role: collaborator

NCT01493973Phase 2Completed

Efficacy Study of Epoetin Alfa in Friedreich Ataxia

Role: collaborator

NCT01339884Phase 1Completed

A Study of Resveratrol as Treatment for Friedreich Ataxia

Role: collaborator

NCT00803868Phase 2Terminated

Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

Role: collaborator

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