Ultra-Rare Disease Studying Subjects Aged 3 Years and Younger

About the Case Study

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:

• Project Management
• Clinical Operations & Trial Management
• Pharmacovigilance
• Quality Assurance

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