Key Insights

Highlights

Success Rate

82% trial completion

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 39/100

Termination Rate

8.7%

4 terminated out of 46 trials

Success Rate

81.8%

-4.7% vs benchmark

Late-Stage Pipeline

11%

5 trials in Phase 3/4

Results Transparency

44%

8 of 18 completed with results

Key Signals

8 with results82% success

Data Visualizations

Phase Distribution

27Total
P 1 (3)
P 2 (19)
P 3 (4)
P 4 (1)

Trial Status

Completed18
Recruiting11
Active Not Recruiting6
Terminated4
Unknown4
Enrolling By Invitation2

Trial Success Rate

81.8%

Benchmark: 86.5%

Based on 18 completed trials

Clinical Trials (46)

Showing 20 of 20 trials
NCT06842355Phase 2RecruitingPrimary

A Study of TYRA-300 in Children With Achondroplasia: BEACH301

NCT07441876Phase 2RecruitingPrimary

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

NCT06732895Phase 2RecruitingPrimary

A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

NCT06079398Phase 2RecruitingPrimary

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

NCT07169279Phase 2RecruitingPrimary

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT02597881RecruitingPrimary

Achondroplasia Natural History Multicenter Clinical Study

NCT06164951Phase 3CompletedPrimary

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

NCT04554940Phase 2Active Not RecruitingPrimary

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

NCT02724228Phase 2Active Not RecruitingPrimary

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

NCT03989947Phase 2Active Not RecruitingPrimary

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03424018Phase 3Active Not RecruitingPrimary

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT07388966RecruitingPrimary

Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia

NCT06433557Phase 2Active Not RecruitingPrimary

A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia

NCT05598320Phase 2CompletedPrimary

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

NCT07301463RecruitingPrimary

A Study in Children With Achondroplasia

NCT07297875Phase 1Not Yet RecruitingPrimary

A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia

NCT06168201RecruitingPrimary

VIrtual STudy in Achondroplasia for the US (VISTA)

NCT05145010Phase 2Enrolling By InvitationPrimary

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05929807Phase 2Enrolling By InvitationPrimary

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

NCT04265651Phase 2CompletedPrimary

Study of Infigratinib in Children With Achondroplasia

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