Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 65/100

Failure Rate

18.9%

99 terminated/withdrawn out of 523 trials

Success Rate

79.1%

-7.4% vs industry average

Late-Stage Pipeline

25%

132 trials in Phase 3/4

Results Transparency

49%

182 of 374 completed trials have results

Key Signals

12 recruiting182 with results77 terminated22 withdrawn

Enrollment Performance

Analytics

Phase 2
160(35.2%)
Phase 1
124(27.3%)
Phase 3
74(16.3%)
Phase 4
58(12.7%)
N/A
38(8.4%)
Early Phase 1
1(0.2%)
455Total
Phase 2(160)
Phase 1(124)
Phase 3(74)
Phase 4(58)
+2 more

Activity Timeline

Global Presence

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Clinical Trials (523)

Showing 20 of 523 trials
NCT00231400Recruiting

Pompe Disease Registry Protocol

Role: lead

NCT00196742Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

Role: lead

NCT00567073Recruiting

Pompe Pregnancy Sub-Registry

Role: lead

NCT00358943Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Role: lead

NCT01665326Recruiting

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

Role: collaborator

NCT06666413Phase 4Recruiting

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Role: lead

NCT04883242Phase 2Active Not Recruiting

Isatuximab, Carfilzomib, Pomalidomide, and Dexamethasone for the Treatment of Relapsed or Refractory Multiple Myeloma

Role: collaborator

NCT05401786Phase 2Active Not Recruiting

Anti-PD-1 Re-challenge After Immune Priming by Ipilimumab and Immune Boosting by Radiotherapy in Advanced NSCLC

Role: collaborator

NCT00144794Completed

Mucopolysaccharidosis I (MPS I) Registry

Role: lead

NCT03019406Phase 2Active Not Recruiting

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Role: lead

NCT04221451Phase 3Terminated

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Role: lead

NCT00625755Phase 1Completed

A Phase I/II Study to Assess the Safety and Efficacy of Vaccinations With Allogeneic Dendritic Cells: Autologous Tumor-Derived Cells Subjected to Electrofusions in Patients With AJCC Stage IV Renal Cell Carcinoma

Role: collaborator

NCT00626860Phase 1Completed

The Use of Dendritic Cell/Tumor Hybridomas as a Novel Tumor Vaccine in Patients With Advance Melanoma

Role: collaborator

NCT03292861Phase 2Enrolling By Invitation

The Effect and Safety Profile of Thymoglobulin® in Primary Cardiac Transplant Recipients

Role: collaborator

NCT07087106Phase 4Recruiting

Glucose Consumption in MS Using [F-18]FDG-PET

Role: collaborator

NCT03754790Phase 3Active Not Recruiting

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Role: lead

NCT06121180Phase 2Recruiting

Study of Cemiplimab Plus Ziv-Aflibercept for Subjects With Metastatic Uveal Melanoma

Role: collaborator

NCT03368664Phase 3Terminated

A Study to Evaluate Efficacy, Safety, and Tolerability of Alemtuzumab in Pediatric Patients With RRMS With Disease Activity on Prior DMT

Role: lead

NCT05247866Phase 4Completed

Efficacy of Dupilumab on Facilitated Food Introduction in Eosinophilic Esophagitis

Role: collaborator

NCT05164055Phase 4Active Not Recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Role: lead