Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

7.5%

5 terminated/withdrawn out of 67 trials

Success Rate

90.0%

+3.5% vs industry average

Late-Stage Pipeline

4%

3 trials in Phase 3/4

Results Transparency

29%

13 of 45 completed trials have results

Key Signals

9 recruiting13 with results

Enrollment Performance

Analytics

Phase 1
6(33.3%)
Phase 2
5(27.8%)
N/A
4(22.2%)
Phase 3
2(11.1%)
Phase 4
1(5.6%)
18Total
Phase 1(6)
Phase 2(5)
N/A(4)
Phase 3(2)
+1 more

Activity Timeline

Global Presence

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Clinical Trials (67)

Showing 20 of 67 trials
NCT01940094Phase 3Completed

The Assessment of Prednisone In Remission Trial - Centers of Excellence Approach

Role: collaborator

NCT02461615Recruiting

A National Registry For Pulmonary Alveolar Proteinosis

Role: collaborator

NCT00668187Recruiting

A Natural History Study of the Gangliosidoses

Role: collaborator

NCT02583672Phase 2Completed

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

Role: collaborator

NCT03056794Recruiting

Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies

Role: collaborator

NCT01067742Terminated

The Natural History of Mucolipidosis Type IV

Role: collaborator

NCT01241305Recruiting

One-Time DNA Study for Vasculitis

Role: collaborator

NCT01193075Recruiting

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Role: collaborator

NCT01581424Recruiting

Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

Role: collaborator

NCT05402384Phase 2Not Yet Recruiting

AVTX-801 D-galactose Supplementation in SLC35A2-CDG

Role: collaborator

NCT02993796Recruiting

Krabbe Disease Global Patient Registry

Role: collaborator

NCT02432560Recruiting

Safety and Durability of Sirolimus for Treatment of LAM

Role: collaborator

NCT01521429Completed

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Role: collaborator

NCT01933724Phase 3Active Not Recruiting

The Assessment of Prednisone In Remission Trial (TAPIR) - Patient Centric Approach

Role: collaborator

NCT00237315Recruiting

Longitudinal Study of Urea Cycle Disorders

Role: collaborator

NCT03052101Completed

Assessment of Safety of Air Travel in Patients With Pulmonary Langerhans Cell Histiocytosis

Role: collaborator

NCT03040115Completed

Assessment of Safety of Air Travel in Patients With Birt-Hogg-Dube Syndrome

Role: collaborator

NCT02232477Not ApplicableTerminated

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Role: collaborator

NCT00266760Completed

Characteristics of Episodic Ataxia Syndrome

Role: collaborator

NCT03064048Not ApplicableCompleted

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

Role: collaborator