Key Insights

Highlights

Success Rate

85% trial completion

Published Results

16 trials with published results (22%)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 39/100

Termination Rate

9.5%

7 terminated out of 74 trials

Success Rate

85.1%

-1.4% vs benchmark

Late-Stage Pipeline

12%

9 trials in Phase 3/4

Results Transparency

40%

16 of 40 completed with results

Key Signals

16 with results85% success

Data Visualizations

Phase Distribution

39Total
Not Applicable (9)
Early P 1 (1)
P 1 (13)
P 2 (7)
P 4 (9)

Trial Status

Completed40
Unknown15
Recruiting8
Terminated7
Active Not Recruiting3
Enrolling By Invitation1

Trial Success Rate

85.1%

Benchmark: 86.5%

Based on 40 completed trials

Clinical Trials (74)

Showing 20 of 20 trials
NCT07123155Phase 2RecruitingPrimary

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT00231400Recruiting

Pompe Disease Registry Protocol

NCT03533673Phase 1CompletedPrimary

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCT01665326RecruitingPrimary

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT06121011RecruitingPrimary

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT04639336RecruitingPrimary

Cognitive and Neurological Pathologies in Pompe Disease

NCT05734521RecruitingPrimary

Avalglucosidase Alfa Pregnancy Study

NCT02675465Phase 1CompletedPrimary

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT03694561Active Not RecruitingPrimary

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03865836UnknownPrimary

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

NCT00688597Phase 2TerminatedPrimary

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT06605612Enrolling By Invitation

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

NCT05943678Active Not RecruitingPrimary

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.

NCT05687474Completed

Baby Detect : Genomic Newborn Screening

NCT01380743Phase 2CompletedPrimary

Drug-drug Interaction Study

NCT05083806Not ApplicableCompletedPrimary

MSOT in Pompe Disease

NCT04093349Phase 1Active Not RecruitingPrimary

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT06539169Recruiting

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT02354664CompletedPrimary

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

NCT00566878Completed

Pompe Lactation Sub-Registry

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Research Network

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