PKU Drug's nutritional impact under the microscope
NCT ID NCT03856203
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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.
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- Newborn screening study aims to catch rare diseases at birth
- PKU diet in childhood may shape adult IQ, study finds
- New PKU drink passes small taste and tolerance test
- New PKU formula shows promise in managing blood levels
- New gene therapy aims to tame PKU in first human test