NCT06780332

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

A Phase 4 Study to Evaluate the Impact of a Rapid Drug Desensitization (RDD) Protocol for Adults With Phenylketonuria and Experiencing Hypersensitivity Reactions to Palynziq

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine if rapid drug desensitization (RDD) to Palynziq will improve drug tolerability and treatment persistence in adult patients on commercial Palynziq experiencing hypersensitivity reactions (HSRs) leading to treatment interruption or reduction of dose or dosing frequency. See Section 10.8 for full list of HSR preferred terms. Study details include: * Study duration: Up to 30 weeks (up to 6 weeks for Screening, then RDD, and 24 weeks of follow-up) * RDD duration: 1 day * Palynziq dosing/follow-up duration: 24 weeks * Palynziq dosing frequency: Individualized

Detailed description

This is a Phase 4 study to evaluate the impact of RDD on adult (≥18 years old) participants with PKU who have experienced HSRs leading to treatment interruption or reduction of dose or dosing frequency while receiving commercial Palynziq. This is not a blinded study. All participants will undergo the same assessments: Screening, the RDD in clinic on Day 1, in clinic dosing on Day 2, followed by remote visits in weeks 2-24 post-RDD to monitor for HSRs and changes in Palynziq dosing. Remote visits will occur every other week from Week 2 through Week 12, then monthly until Week 24. The total duration of participation in the trial will be approximately 30 weeks: up to 6 weeks for screening, followed by RDD and 24 weeks of post-RDD follow-up. No Data Monitoring Committee (DMC) will be used for this trial.

Conditions

• Phenylketonuria
• PKU

Interventions

Type	Name	Description
DRUG	RDD to Palynziq	Delivery of gradually increasing doses of Palynziq at fixed time intervals over the course of several hours to a cumulative dose equal to the reactive dose. Reactive dose is defined as the dose at which the reactive HSR occurred which led to treatment interruption or reduction of dose or dose frequency.

Timeline

• Start date: 2025-02-07
• Primary completion: 2026-08-01
• Completion: 2026-08-01
• First posted: 2025-01-17
• Last updated: 2026-04-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study