Condition

Phenylketonuria (PKU) clinical trials and research

• 8Clinical trials
• 9Countries

Newest Phenylketonuria (PKU) clinical trials

All recruiting trials (8)

• How often to check phenylalanine in adults with classic PKU

  This study tests whether checking phenylalanine levels weekly instead of monthly helps adults with classic PKU keep their levels under better control.

  Recruiting Jagiellonian University · Krakow · NCT07825883

• MZE782 in adults with phenylketonuria (PKU) testing safety and efficacy

  This study tests whether MZE782 works safely for adults with PKU compared with a placebo.

  Recruiting at 9 sites Phase 2 Maze Therapeutics · Oakland, California and 8 other locations · NCT07694440

• PL54 injection safety in adults with PKU

  This study tests whether PL54 is safe and tolerable when given once or in multiple weekly shots to adults with phenylketonuria.

  Recruiting Phase 1 Chongqing Peg-Bio Biopharm Co., Ltd. · Hefei, Anhui · NCT07672756

• Multiomics (genome plus metabolome) approach for adults with phenylketonuria

  This project will test whether combining whole-genome sequencing from saliva and non-targeted blood metabolomics can find genetic and metabolic patterns linked to symptoms in adults who were treated for phenylketonuria from birth.

  Recruiting University Hospital, Tours · Angers and 14 other locations · NCT07484945

• Investigating brain aging in adults with phenylketonuria

  This study looks at how living with phenylketonuria (PKU) affects brain aging and thinking skills in adults who have followed a special diet since they were babies.

  Recruiting Insel Gruppe AG, University Hospital Bern · Bern · NCT06969209

• Long-term JNT-517 treatment for people with phenylketonuria

  This trial tests the long-term safety of JNT-517 in children and adults with PKU, including people who previously took JNT-517 and those who are new to the drug.

  Recruiting at 12 sites Phase 3 Otsuka Pharmaceutical Development & Commercialization, Inc. · Gainesville, Florida and 11 other locations · NCT06628128

• Long-term evaluation of immune response in PKU patients receiving Palynziq

  This study is testing how well Palynziq works over 10 years for people with PKU by looking at their immune responses and overall health.

  Recruiting at 11 sites BioMarin Pharmaceutical · Aurora, Colorado and 10 other locations · NCT06305234

• Long-term safety assessment of pegvaliase in patients with PKU

  This study is testing the long-term safety of pegvaliase in people with PKU to see how well it works and if there are any side effects over time.

  Recruiting at 26 sites BioMarin Pharmaceutical · Aurora, Colorado and 25 other locations · NCT05813678

Quick answers

How many Phenylketonuria (PKU) clinical trials are recruiting right now?

8, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 9 countries. The most sites are in United States (4 trials), France (2 trials), Germany (1 trial), Italy (1 trial), Australia (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 1, Phase 2: 1, Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →