CompletedNot Applicable

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

United States12 participantsStarted 2019-03-08

Plain-language summary

Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosis of phenylketonuria * Aged 18-65 years * Following a protein-restricted diet with or without medical food within the last 30 days * Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day). * If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins. * Within ±90 days of starting treatment with pegvaliase at time of study enrollment Exclusion Criteria: * Unable to consent to study * Under age 18 or over age 65 years * Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days. * Females who are currently pregnant or lactating

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.

Timeframe: 15 months

Intra-subject change in serum markers of protein status at months 0, 9, and 15.

Timeframe: 15 months

Intra-subject difference in lean body mass (%LBM) from month 0 to 15.

Timeframe: 15 months

Intra-subject difference in bone mineral density from month 0 to 15.

Timeframe: 15 months

Trial details

NCT IDNCT03856203

SponsorBoston Children's Hospital

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2022-12-31

View on ClinicalTrials.gov More Phenylketonurias trials

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.

Timeframe: 15 months

Intra-subject change in serum markers of protein status at months 0, 9, and 15.

Timeframe: 15 months

Intra-subject difference in lean body mass (%LBM) from month 0 to 15.

Timeframe: 15 months

Intra-subject difference in bone mineral density from month 0 to 15.

Timeframe: 15 months