Table 1.
RDCRN PRISM Protocols
| Protocol | Consortium | Site Locations | Target Accrual | Study Type | Intervention Type | Age | Disease Status |
|---|---|---|---|---|---|---|---|
| Abatacept (CTLA4-Ig) for the Treatment of Relapsing, Non-Severe, Granulomatosis with Polyangiitis (ABROGATE) | Vasculitis Clinical Research Consortium (VCRC) | US, Canada, UK, Ireland, Germany | 66 |
Interventional RCT, Phase III |
• Double-blinded • Placebo-controlled • Investigational agent |
15 years old and up | Mild flare- active disease at enrollment |
| A Randomized, Multicenter Study for Isolated Skin Vasculitis (ARAMIS) | Vasculitis Clinical Research Consortium (VCRC) | US, Canada | 90 |
Interventional sequential multiple assignment RCT |
• 3 standard of care medications | 18 years old and up | Active disease at enrollment |
| Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis (LongPAP) | Rare Lung Disease Consortium (RLDC) | US | 100 | Longitudinal, Observational | • None | All ages | Active Disease/ Remission (no major disease activity) |
| Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects with Chronic Hepatitis C | Porphyrias Consortium (PC) | US | 49 | Interventional |
• Open label • One arm |
18 years old and up | PCT with chronic Hepatitis C |
| A Randomized Double-Blind Controlled Trial of Everolimus in Individuals with PTEN Mutations | Developmental Synaptopathies Consortium (DSC) | US | 40 |
Interventional, Phase I/II |
• Placebo-controlled • Investigational agent |
5–45 years old | Outpatients with PTEN genetic mutation |
| A Prospective, Multicenter Study to Compare and Validate Endoscopic, Histologic, Molecular, and Patient-Reported Outcomes in Pediatric and Adult Patients with Eosinophilic Esophagitis, Gastritis, and Colitis | Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) | US | 1050 | Observational | • None | 3 years old and up | Active disease at enrollment |