Pegvaliase for Phenylketonuria
(PEGASUS Trial)

Recruiting in Palo Alto (17 mi)

+21 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: BioMarin Pharmaceutical

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing pegvaliase injections in adolescents with PKU. The goal is to see if these injections are safe and effective. Pegvaliase helps the body break down a harmful substance that people with PKU cannot process on their own. Pegvaliase is a newer treatment for PKU, following earlier treatments like dietary restriction and sapropterin.

Eligibility Criteria

This trial is for adolescents aged 12-17 in the US (or 12-15 in the EU) with Phenylketonuria (PKU) who haven't been able to control their blood Phe levels through current treatments. They must be stable on any psychiatric meds, willing to follow a diet plan, and have an adult supervisor during treatment. Sexually active participants need to use two forms of contraception.

Inclusion Criteria

I am using two forms of birth control and do not plan to get pregnant or father a child while in the study.

I've been on a stable dose of my psychiatric medication for at least 8 weeks and can keep it steady.

I am able to understand and sign the consent form.

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Exclusion Criteria

Your creatinine levels in your blood are higher than 1.5 times the normal upper limit.

I haven't used any PEG-containing injectable drugs recently, except for COVID-19 vaccines.

I have had an organ transplant or am on long-term immune-weakening medication.

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Treatment Details

Interventions

Pegvaliase (Enzyme substitution therapy)

Trial OverviewThe study tests Pegvaliase injections against a diet-only approach in managing PKU. It's an open-label Phase 3 trial where about 54 young people are randomly assigned to either get the drug or stick with dietary management to see which is safer and works better.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: PegvaliaseExperimental Treatment1 Intervention

Group II: Drug: Diet OnlyExperimental Treatment1 Intervention

Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72, initiating pegvaliase treatment beginning Week 73 and, from Weeks 73 through 215.