Pharm-Olam was contracted to execute a multicenter, pre-Phase I non-interventional study centered on young children, aged 3 years and younger, who have an Ultra-Rare Disease. There was no investigational product to evaluate. It was a natural history study designed to clinically assess disease progression in patients and the burden of the condition as it relates to caregivers. This natural history study has acted as a critical dataset for driving the future of their development program and future clinical studies.
Program Overview: Pre-Phase 1
Indication: Multicenter, Non-interventional Natural History Study to Observe Young Pediatric Subjects with an Ultra Rare Disease (1 in 50,000 newborns)
Sites & Locations: 8 sites, 5 countries
Services Provided:
