Key Insights

Highlights

Success Rate

100% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 50/100

Termination Rate

0.0%

0 terminated out of 43 trials

Success Rate

100.0%

+13.5% vs benchmark

Late-Stage Pipeline

9%

4 trials in Phase 3/4

Results Transparency

10%

2 of 20 completed with results

Key Signals

2 with results100% success

Data Visualizations

Phase Distribution

29Total
Not Applicable (14)
P 1 (6)
P 2 (5)
P 3 (4)

Trial Status

Completed20
Recruiting11
Unknown5
Enrolling By Invitation3
Active Not Recruiting2
Not Yet Recruiting1

Trial Success Rate

100.0%

Benchmark: 86.5%

Based on 20 completed trials

Clinical Trials (43)

Showing 20 of 20 trials
NCT06667453Phase 2RecruitingPrimary

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

NCT07008469Phase 3Enrolling By Invitation

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07220603Phase 2RecruitingPrimary

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

NCT05027269Phase 1Completed

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT06204809Phase 1CompletedPrimary

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

NCT06300307Phase 1RecruitingPrimary

Study of ATX-01 in Participants With DM1

NCT07385443RecruitingPrimary

The Spanish National Registry for Myotonic Dystrophy Type 1

NCT06411288Phase 3Active Not Recruiting

Global Study of Del-desiran for the Treatment of DM1

NCT07075965Phase 1Not Yet RecruitingPrimary

Calcium Channel Blocker in Myotonic Dystrophy Type 1

NCT05532813Phase 3Recruiting

Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)

NCT06138743Phase 1RecruitingPrimary

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

NCT05479981Phase 2Completed

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT06101940Enrolling By InvitationPrimary

A Multicenter Phenotype-Genotype Analysis of DM1 Patients in China

NCT03424460Not ApplicableCompletedPrimary

Venous Thromboembolism in Myotonic Dystrophy Type 1

NCT02858908Phase 2CompletedPrimary

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

NCT07136844Not ApplicableRecruiting

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

NCT05865483CompletedPrimary

Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)

NCT07072676Not ApplicableEnrolling By Invitation

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

NCT03981575RecruitingPrimary

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

NCT04656210Active Not RecruitingPrimary

Myotonic Dystrophy - Vascular and Cognition

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