Longitudinal Natural History of Disorders Associated with Hyperphenylalaninemia

NIH RePORTER · NIH · U54 · $970,549 · view on reporter.nih.gov ↗

Abstract

4. PROJECT SUMMARY – PROJECT 1 The objective of Clinical Project #1 is to develop a comprehensive longitudinal natural history study to capture outcomes data throughout the lifespan on individuals with inborn errors of metabolism causing hyperphenylalaninemia (elevated blood phenylalanine (Phe)). Eligible subjects will include individuals of all ages of either gender with molecularly proven phenylalanine hydroxylase (PAH) deficiency, a deficiency of biopterin synthesis or recycling (including GTP cyclohydrolase I, 6-pyruvoyl-tetrahydropterin synthase (PTPS), dihydropteridine reductase (DHPR) or pterin-4a-carbinolamine dehydratase (PCD) deficiencies), or deficiency of the PAH co-chaperone protein DNAJC12. This project will comprehensively and longitudinally evaluate the health, neurologic, cognitive, neuropsychiatric, patient-reported, and quality-of-life outcomes in a large cohort of individuals with these inborn errors of metabolism and explore the interrelationships between outcome and blood Phe levels, or other biomarkers. The results of this study will allow refinement and improvement of current and future therapies for the most common inborn error of metabolism and the rarer conditions associated with hyperphenylalaninemia.

Key facts

NIH application ID
10019406
Project number
5U54HD100982-02
Recipient
OREGON HEALTH & SCIENCE UNIVERSITY
Principal Investigator
GEORGIANNE L ARNOLD
Activity code
U54
Funding institute
NIH
Fiscal year
2020
Award amount
$970,549
Award type
5
Project period
2019-09-16 → 2024-08-31