Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

Launched by BIOMARIN PHARMACEUTICAL · Jan 13, 2025

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ClinConnect Summary

This clinical trial is studying a method called rapid drug desensitization (RDD) to help adults with a condition called phenylketonuria (PKU) who have experienced allergic reactions to a medication called Palynziq. The goal is to see if RDD can make it easier for these patients to tolerate Palynziq, allowing them to continue their treatment without interruptions or needing to decrease their doses. The study will last up to 30 weeks, with one day for the desensitization process and 24 weeks of follow-up to monitor how well the patients do on Palynziq afterward.

To be eligible for the trial, participants must be 18 years or older, have PKU, and have had significant allergic reactions to Palynziq that caused them to stop or reduce their treatment. Both men and women can take part, and they must be able to provide informed consent. Throughout the study, participants will undergo a quick desensitization procedure and then take Palynziq as directed. It’s important to note that those with certain medical conditions, such as uncontrolled asthma or severe allergic reactions, may not be eligible. This trial aims to improve the experience of taking Palynziq for those who have struggled with allergic reactions.

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Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• * 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.
• • Other Exclusions 9. Participants must not be experiencing reactions that are not appropriate for RDD, including severe cutaneous adverse reactions (SCAR) as defined by the American Academy for Allergy, Asthma, and Immunology.