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 |
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Recruitment Status :
Recruiting
First Posted : October 14, 2022
Last Update Posted : May 3, 2024
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Sponsor:
BioMarin Pharmaceutical
Collaborator:
Syneos Health
Information provided by (Responsible Party):
BioMarin Pharmaceutical
| Tracking Information | |||||||
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| First Submitted Date | October 11, 2022 | ||||||
| First Posted Date | October 14, 2022 | ||||||
| Last Update Posted Date | May 3, 2024 | ||||||
| Actual Study Start Date | November 22, 2022 | ||||||
| Estimated Primary Completion Date | October 4, 2032 (Final data collection date for primary outcome measure) | ||||||
| Current Primary Outcome Measures |
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.
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| Original Primary Outcome Measures | Same as current | ||||||
| Change History | |||||||
| Current Secondary Outcome Measures |
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| Original Secondary Outcome Measures |
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| Current Other Pre-specified Outcome Measures | Not Provided | ||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||
| Descriptive Information | |||||||
| Brief Title | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||||||
| Official Title | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||||||
| 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. | ||||||
| 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 Type | Observational | ||||||
| Study Design | Observational Model: Cohort Time Perspective: Prospective |
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| Target Follow-Up Duration | Not Provided | ||||||
| Biospecimen | Not Provided | ||||||
| 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 | ||||||
| Condition | Phenylketonuria, Maternal | ||||||
| Intervention | Drug: Pegvaliase
Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.
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| Study Groups/Cohorts | 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.
Intervention: Drug: Pegvaliase
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| Publications * | Not Provided | ||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||
| Recruitment Status | Recruiting | ||||||
| Estimated Enrollment |
50 | ||||||
| Original Estimated Enrollment | Same as current | ||||||
| Estimated Study Completion Date | October 4, 2032 | ||||||
| Estimated Primary Completion Date | October 4, 2032 (Final data collection date for primary outcome measure) | ||||||
| Eligibility Criteria | Inclusion Criteria:
Exclusion Criteria: • Currently participating in a BioMarin sponsored interventional study of any investigational product, device, or procedure |
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| Sex/Gender |
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| Ages | Child, Adult, Older Adult | ||||||
| Accepts Healthy Volunteers | Not Provided | ||||||
| Contacts |
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| Listed Location Countries | Germany, Italy, United States | ||||||
| Removed Location Countries | |||||||
| Administrative Information | |||||||
| NCT Number | NCT05579548 | ||||||
| Other Study ID Numbers | 165-504 | ||||||
| Has Data Monitoring Committee | No | ||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement | Not Provided | ||||||
| Current Responsible Party | BioMarin Pharmaceutical | ||||||
| Original Responsible Party | Same as current | ||||||
| Current Study Sponsor | BioMarin Pharmaceutical | ||||||
| Original Study Sponsor | Same as current | ||||||
| Collaborators | Syneos Health | ||||||
| Investigators |
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| PRS Account | BioMarin Pharmaceutical | ||||||
| Verification Date | May 2024 | ||||||