RECRUITING

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2025-01

Study Completion:2025-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06780332

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Age: 1.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.	* Medical Conditions: 1. Participant may not have any kind of disorders that, in the opinion of the investigator, may compromise the ability of the participant to give written informed consent and/or comply with any aspect of the study, and/or where any aspect of participation would be medically inadvisable for the underlying disorder. 2. Participant must not be pregnant on the day of the RDD. 3. Participants with uncontrolled asthma, active upper repiratory infection or other active infections, or cardiovascular disease. Asthma control will be assessed using the Asthma Control Test at screening. * Prior/Concomitant Therapies: 4. Participants were not using antihistamine premedication(s) at the time of reactive HSRs. 5. Participants are willing and able to resume and tolerate Palynziq at the reactive dose prior to the RDD. 6. Participant must not be receiving concurrent injectables containing PEG with the exception of PEG-containing vaccines, such as COVID-19 vaccinations (see Section 6.7). 7.Participants receiving beta blockers. * Prior/Concurrent Clinical Study Experience 8. Participants may not be currently participating in an interventional study of any investigational product, device, or procedure, or any other BioMarin clinical trial or post-marketing study.

Inclusion Criteria

Exclusion Criteria

* Age:
1.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.

* Medical Conditions:
1. Participant may not have any kind of disorders that, in the opinion of the investigator, may compromise the ability of the participant to give written informed consent and/or comply with any aspect of the study, and/or where any aspect of participation would be medically inadvisable for the underlying disorder.
2. Participant must not be pregnant on the day of the RDD.
3. Participants with uncontrolled asthma, active upper repiratory infection or other active infections, or cardiovascular disease. Asthma control will be assessed using the Asthma Control Test at screening.
* Prior/Concomitant Therapies:
4. Participants were not using antihistamine premedication(s) at the time of reactive HSRs.
5. Participants are willing and able to resume and tolerate Palynziq at the reactive dose prior to the RDD.
6. Participant must not be receiving concurrent injectables containing PEG with the exception of PEG-containing vaccines, such as COVID-19 vaccinations (see Section 6.7).
7.Participants receiving beta blockers.
* Prior/Concurrent Clinical Study Experience 8. Participants may not be currently participating in an interventional study of any investigational product, device, or procedure, or any other BioMarin clinical trial or post-marketing study.

Contacts and Locations

Study Contact

165-402 Trial Specialist

CONTACT

1-800-983-4587

medinfo@bmrn.com

165-402 Medical Director, MD

CONTACT

1-800-983-4587

medinfo@bmrn.com

Principal Investigator

Medical Director, MD

STUDY_DIRECTOR

BioMarin Pharmaceutical

Study Locations (Sites)

Uncommon Cures

Chevy Chase, Maryland, 20815

United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

Medical Director, MD, STUDY_DIRECTOR, BioMarin Pharmaceutical

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-01

Study Completion Date2025-11

Study Record Updates

Study Start Date2025-01

Study Completion Date2025-11

Terms related to this study

Keywords Provided by Researchers

Phenylketonuria
PKU
Palynziq
pegvaliase

Additional Relevant MeSH Terms

Phenylketonuria
PKU