Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)
Phenylketonurias
About this trial
This is an interventional treatment trial for Phenylketonurias focused on measuring N-of-1 randomized controlled trials
Eligibility Criteria
Inclusion Criteria:
- age ≥ 18 years
- diagnosis of classical PKU (blood phenylalanine ≥1200 µmol pre-treatment or off-treatment
- ≥ 2 PKU-related symptoms with clinically meaningful negative impact on daily life
- is in regular clinical monitoring and is treated with a PKU diet with a natural protein restriction and medical foods
- average blood phenylalanine levels between 360 and 900 µmol in past one year
- able and willing to provide consent
- demonstrates capacity to complete all requirements of the protocol
- if on medications approved by the Principal Investigator agrees not to alter dose for duration of study
- has stable daily access to phone, internet, and physical address
Exclusion Criteria:
- women who are breastfeeding, pregnant or planning to become pregnant in the next year
- use of adjuvant PKU treatments (LNAAs, Kuvan®, Palinziq®) within past 3 months
- use of psychotropic medications, melatonin, or any serotonin-reuptake inhibitors within past 3 months
- demonstrates insufficient motivation or time required to complete full trial
Sites / Locations
- University of Southern CaliforniaRecruiting
Arms of the Study
Arm 1
Arm 2
Active Comparator
Placebo Comparator
Active LNAA
Inactive LNAA
Active LNAA tablets are given to each participant in 3 multiple crossovers for a total exposure to the Active Comparator 3 times. The allocation is randomized within each cycle of two treatments (active/inactive). There are 3 total cycles for each participant. The intervention is PreKUnil® tablets.
Inactive LNAA tablets (placebos) are given to each participant in 3 multiple crossovers for a total exposure to the Inactive Comparator 3 times. The allocation is randomized within each cycle of two treatments (inactive/active). There are 3 total cycles for each participant. The placebo intervention is PreKUnil® placebo tablets.