This study is currently not recruiting participants.

An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/day Dose) in Adults with Phenylketonuria

Investigating the Safety and Efficacy of Higher Doses of Investigational Medication for Phenylketonuria (PKU)

Not Recruiting
100 years or below
All
Phase N/A

Brief description of study.

PALYNZIQ was approved by the United States FDA in May 2018 for daily doses up to 40 mg after dose escalation as directed by the prescribing healthcare provider experienced in the management of PKU. Pegvaliase has not been approved as safe and effective by the FDA at doses above 40 mg/day. This study will help learn more about whether the study drug at doses above 40 mg/day is safe and whether it works in lowering levels of Phe that is in blood.

Detailed description of study

PALYNZIQ was approved by the United States FDA in May 2018 for daily doses up to 40 mg after dose escalation as directed by the prescribing healthcare provider experienced in the management of PKU. Pegvaliase has not been approved as safe and effective by the FDA at doses above 40 mg/day. This study will help learn more about whether the study drug at doses above 40 mg/day is safe and whether it works in lowering levels of Phe that is in blood.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: phenylketonuria
  • Age: 100 years or below
  • Gender: All

This study investigates the safety and effectiveness of an investigational medication for individuals with Phenylketonuria (PKU), a genetic disorder that increases the levels of a substance called phenylalanine (Phe) in the blood. The focus is on doses above 40 mg per day, which have not yet been approved by the FDA for safety and effectiveness.

Participants in this study will receive doses of the investigational medication above 40 mg per day. The study aims to monitor the effects on blood Phe levels and observe any potential side effects.

  • Who can participate: Eligibility criteria are not provided in the source document. However, typical criteria might include age, diagnosis of PKU, and specific health conditions. Please refer to the study contact for detailed eligibility information.
  • Study details: Participants will receive doses of the investigational medication that are higher than the standard 40 mg per day to assess safety and effectiveness in lowering Phe levels.
Updated on 19 Feb 2024. Study ID: 1806154271

Interested in the study?

Select a study center that’s convenient for you, and get in touch with the study team.

Connect with the study team