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A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding (PALomino)

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ClinicalTrials.gov Identifier: NCT05579548
Recruitment Status : Recruiting
First Posted : October 14, 2022
Last Update Posted : January 5, 2024
Sponsor:
Collaborator:
Syneos Health
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Description
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Brief Summary:
This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.

Condition or disease Intervention/treatment
Phenylketonuria, Maternal Drug: Pegvaliase

Detailed Description:
Women prescribed pegvaliase by their healthcare provider (HCP) who become pregnant during treatment may decide to participate in the study and enroll via a centralized call center model. Upon consent, data will be collected from the patient's HCP(s) retrospectively for at least 3 months prior to Last Menstrual Period (LMP). Data will be collected during pregnancy and throughout the infant's first year of life. Pegvaliase exposure will be recorded during pregnancy and breastfeeding including exposure during each trimester of pregnancy. Duration of individual subject participation will be up to approximately 21 months.
Study Design
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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Actual Study Start Date : November 22, 2022
Estimated Primary Completion Date : October 4, 2032
Estimated Study Completion Date : October 4, 2032

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Pregnant Women with PKU
This study will enroll pregnant women diagnosed with PKU who have been treated with pegvaliase from 2 weeks prior to LMP or at any time during pregnancy.
 Drug: Pegvaliase
Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.


Outcome Measures
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Primary Outcome Measures :
  1. Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period [ Time Frame: 10 years ]
    To estimate the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) among subjects with PKU treated with pegvaliase during pregnancy and fetal/infant outcomes (all major congenital malformations [MCMs] and specifically microcephaly and congenital heart defects], FGR, small for gestational age [SGA], low birth weight, preterm birth, failure to thrive, and developmental delays) among their offspring exposed to pegvaliase during pregnancy.


Secondary Outcome Measures :
  1. Pregnancy Outcomes [ Time Frame: 10 years ]
    To compare the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) and fetal/infant outcomes (all MCMs and specifically microcephaly and congenital heart defects, FGR, SGA, low birth weight, preterm birth, failure to thrive, and developmental delays) among subjects with PKU treated with pegvaliase during pregnancy and their offspring to information on those same outcomes in non-pegvaliase exposed, pregnant women with PKU as described in reference literature

  2. Pregnancy Outcomes [ Time Frame: 10 years ]
    To examine differences in the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) and fetal/infant outcomes (all MCMs and specifically microcephaly and congenital heart defects, FGR, SGA, low birth weight, preterm birth, failure to thrive, and developmental delays) among subjects with PKU treated with pegvaliase during pregnancy and their offspring by maternal blood Phe levels

  3. Serious Adverse Events [ Time Frame: 10 years ]
    To estimate the frequency of serious adverse events (SAEs) other than CMs in infants exposed to pegvaliase during pregnancy through their first year of life

  4. Maternal Pegvaliase use during breastfeeding [ Time Frame: 10 years ]
    To estimate the frequency of selected outcomes in subjects with PKU treated with pegvaliase during breastfeeding (low milk supply) and their infants (failure to thrive and SAEs) through their first year of life


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Only pregnant women with PKU who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period are eligible.
Sampling Method:   Probability Sample
Study Population
Pregnant women with PKU who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period
Criteria

Inclusion Criteria:

  • Subject (or a legally authorized representative) consent obtained prior to enrollment. Consent will be obtained in compliance with any country-specific regulations or requirements
  • Confirmation of ongoing pregnancy. Subjects with unknown outcomes will be classified as prospective pregnancies; subjects who have undergone prenatal testing (eg, targeted ultrasound, amniocentesis) regardless of findings will be classified as retrospective pregnancies
  • Diagnosed with PKU per local standard of care
  • Documentation that the subject was treated with pegvaliase at any point starting from 2 weeks prior to the date of LMP
  • Agrees to permit the Investigator (ie, CRP, CCA, PI) to contact the subject's HCPs (eg, PCP, PKU-treating physician, OB, nurse, midwife) and the infant's HCP (eg, pediatrician, neonatologist) for medical information

Exclusion Criteria:

• Currently participating in a BioMarin sponsored interventional study of any investigational product, device, or procedure

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05579548


Contacts
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Contact: 165-504 Program Director 1-800-983-4587 medinfo@bmrn.com

Locations
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United States, North Carolina
Syneos Health Recruiting
Wilmington, North Carolina, United States, 28403
Contact: Coordinating Center    833-672-2203    PalynziqPregnancyStudy@syneoshealth.com   
Germany
Universitaetsklinikum Hamburg Eppendorf Not yet recruiting
Hamburg, Germany, 20246
Contact: Professor Muntau       muntau@uke.de   
Italy
Ospedale San Paolo Not yet recruiting
Milano, Italy, 20142
Contact: Dr. Rovelli       valentina.rovelli@asst-santipaolocarlo.it   
Sponsors and Collaborators
BioMarin Pharmaceutical
Syneos Health
Investigators
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Study Director: Medical Director, MD BioMarin Pharmaceutical
More Information
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT05579548    
Other Study ID Numbers: 165-504
First Posted: October 14, 2022    Key Record Dates
Last Update Posted: January 5, 2024
Last Verified: January 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by BioMarin Pharmaceutical:
Phase 4
Observational
Maternal
 Pregnancy Outcomes
Exposure
Breastfeeding Outcomes
Additional relevant MeSH terms:
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Phenylketonurias
Phenylketonuria, Maternal
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Pregnancy Complications
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases