Ignite Creation Date:
2025-12-24 @ 9:23 PM
Ignite Modification Date:
2025-12-29 @ 3:43 PM
Study NCT ID:
NCT06780332
Status:
RECRUITING
Last Update Posted:
2025-01-23
First Post:
2025-01-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
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:
RECRUITING
Status Verified Date:
2025-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PALLADIUM
Brief 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
* 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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: