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
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Age:

⁃ Participants must be 18 years of age or older inclusive, at the time of signing the informed consent.

• Type of Participant and Disease Characteristics 2. Participants must have phenylketonuria and must have been receiving commercial Palynziq and enrolled in the REMS.

• 3\. Participants must have developed HSRs leading to treatment interruption or reduction of dose or dosing frequency (Graade 2 or above) while on Palynziq, and must be able to undergo RDD within 6weeks from the reactive HSR and last Palynziq dose.

• Sex and Contraceptive/Barrier Requirement 4. Males and females are eligible to participate in this clinical study. Contraceptive use is not required during the study. Please refer to the Pregnancy section of USPI for further information.

• Informed Consent 5. Participants must be capable of giving written informed consent as described in Section 10.1, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Locations
United States
Maryland
Uncommon Cures
RECRUITING
Chevy Chase
Contact Information
Primary
165-402 Trial Specialist
medinfo@bmrn.com
1-800-983-4587
Backup
165-402 Medical Director, MD
medinfo@bmrn.com
1-800-983-4587
Time Frame
Start Date: 2025-01
Estimated Completion Date: 2025-11
Participants
Target number of participants: 10
Treatments
Experimental: RDD
Participants undergoing RDD to Palynziq
Related Therapeutic Areas
Sponsors
Leads: BioMarin Pharmaceutical

This content was sourced from clinicaltrials.gov