Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

6.2%

17 terminated/withdrawn out of 275 trials

Success Rate

92.1%

+5.6% vs industry average

Late-Stage Pipeline

1 trials in Phase 3/4

Results Transparency

17%

34 of 199 completed trials have results

Key Signals

38 recruiting34 with results12 terminated5 withdrawn

Enrollment Performance

Analytics

N/A

61(68.5%)

Phase 1

17(19.1%)

Phase 2

9(10.1%)

Early Phase 1

1(1.1%)

Phase 3

1(1.1%)

89Total

N/A(61)

Phase 1(17)

Phase 2(9)

Early Phase 1(1)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (275)

Showing 20 of 275 trials

NCT06948110Recruiting

Deciphering the Genetic Architecture of Autoimmune Diseases

Role: lead

NCT03854318Recruiting

Longitudinal Studies of Patient With FPDMM

Role: lead

NCT01087320Recruiting

Genome Medical Sequencing for Gene Discovery

Role: lead

NCT05657405Recruiting

Observational Study of Advanced Data Analytics in Genetic Conditions

Role: lead

NCT06595940Recruiting

Genetic Analysis of Uncommon Disease Presentations in Non-US Populations

Role: lead

NCT00001215Enrolling By Invitation

Genetic Studies of Lysosomal Storage Disorders

Role: lead

NCT06664814Phase 2Recruiting

An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis

Role: lead

NCT06092346Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Role: lead

NCT06552754Not ApplicableRecruiting

Cybersickness Prevention and Mitigation in Virtual Reality for Healthy Volunteers

Role: lead

NCT01780168Recruiting

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

Role: lead

NCT01905865Completed

The Role of Uncertainty in Coping: The Experience of Parents of Children With Undiagnosed Medical Conditions

Role: lead

NCT01498263Completed

Inherited Diseases, Caregiving, and Social Networks

Role: lead

NCT05007990Recruiting

Caregiving Networks Across Disease Context and the Life Course

Role: lead

NCT00078078Recruiting

Clinical and Laboratory Study of Methylmalonic Acidemia

Role: lead

NCT00005909Recruiting

Study of Alkaptonuria

Role: lead

NCT00359684Recruiting

Use of Cysteamine in the Treatment of Cystinosis

Role: lead

NCT00837122Completed

Genetics of Type 2 Diabetes in West Africans

Role: lead

NCT01417533Completed

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

Role: lead

NCT02156102Completed

Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)

Role: lead

NCT01952171Completed

The Genetic Basis of Congenital Heart Disease in Africa

Role: lead