Office of Rare Diseases (ORD)
44
4
7
29
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 45/100
4.5%
2 terminated/withdrawn out of 44 trials
93.5%
+7.0% vs industry average
7%
3 trials in Phase 3/4
34%
10 of 29 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (44)
The Assessment of Prednisone In Remission Trial - Centers of Excellence Approach
Role: collaborator
A Randomized Multicenter Study for Isolated Skin Vasculitis
Role: collaborator
One-Time DNA Study for Vasculitis
Role: collaborator
VCRC Tissue Repository
Role: collaborator
RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
Role: collaborator
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
Role: collaborator
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
Role: collaborator
Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations
Role: collaborator
The Assessment of Prednisone In Remission Trial (TAPIR) - Patient Centric Approach
Role: collaborator
Efficacy and Safety of Sirolimus in LAM
Role: collaborator
Characteristics of Episodic Ataxia Syndrome
Role: collaborator
Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa
Role: collaborator
Determining Disease Activity Biomarkers in Individuals With Giant Cell Arteritis
Role: collaborator
Determining Disease Activity Biomarkers in Individuals With Takayasu's Arteritis
Role: collaborator
Longitudinal Protocol for Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
Role: collaborator
Characteristics of Eosinophilic Gastritis, Enteritis, and Colitis in a Multi-Site Cohort
Role: collaborator
CEGIR 7807: Validation of Online Cohort of EGID Patients Enrolled in RDCRN CEGIR Contact Registry
Role: collaborator
Biobanking of Rett Syndrome and Related Disorders
Role: collaborator
CEGIR 7808: Use of Unsedated Transnasal Esophagoscopy (TNE) to Monitor Dietary Management of Eosinophilic Esophagitis in Children
Role: collaborator
Natural History Study of SCID Disorders
Role: collaborator