Experimental enzyme injection aims to tame PKU's toxic amino acid
NCT ID NCT07672756
First seen Jun 29, 2026 · Last updated Jul 02, 2026 · Updated 3 times
Summary
This early-stage trial tests an experimental drug called PL54 in adults aged 18 to 55 with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down the amino acid phenylalanine. PL54 is an enzyme designed to help lower high blood phenylalanine levels, which can cause brain damage if uncontrolled. The study will give participants either a single injection or weekly injections for four weeks, monitoring safety and how well the drug works at different doses.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- PL54 (PEGylated Recombinant Phenylalanine Ammonia-Lyase)
- What this could lead to
- If safe and effective, PL54 could offer a new treatment option for adults with PKU, potentially helping control blood phenylalanine levels without strict diet alone.
- What could go wrong
- This is a very early Phase 1 trial with only 48 participants, focused on safety and dosing. It is too small to prove effectiveness, and side effects or lack of benefit are possible.
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
-
The First Affiliated Hospital of Anhui Medical University
RECRUITINGHefei, Anhui, 230022, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Hidden weight problem: PKU adults face higher obesity risk, scientists investigate why
- New oral drug aims to tame PKU's toxic protein buildup
- Tiny tablets could make PKU management easier for patients
- New PKU drug JNT-517 moves to Long-Term safety trial
- New pill for PKU shows promise in small teen trial
- 10-Year study to track immune risks of PKU drug