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PKU Drug's nutritional impact under the microscope

NCT ID NCT03856203

Knowledge-focused Sponsor: Boston Children's Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 12 adults with PKU to see how their nutrition changed before and during treatment with pegvaliase (Palynziq). Researchers measured protein intake, body composition, bone density, and vitamin levels over 15 months. The goal was to understand if the drug affects nutritional health, not to test its effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pegvaliase (Palynziq)
What this could lead to
If successful, this study could help doctors understand how pegvaliase affects nutrition in people with PKU, guiding better dietary management.
What could go wrong
This is a small, observational study with only 12 participants, so results may not apply to everyone. It does not test whether pegvaliase works or is safe.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

12 people

The number who actually took part.

Started

Mar 2019

Finished

Oct 2023

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

This study will recruit adults with PKU who are starting therapy with pegvaliase and currently following a protein-restricted diet with or without medical food.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Phenylketonurias

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.