Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation

Odevixibat Pregnancy and Lactation Surveillance Program: A Post-marketing, Long-term, Observational, Descriptive Study to Assess the Risk of Pregnancy and Maternal Complications and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Odevixibat During Pregnancy and/or Lactation

The participants of this study will be of any age who are exposed to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and/or at any time during lactation (up to 12 months of infant age or weaning, whichever comes first.

This study will collect data obtained via a variety of sources, including enrolled pregnant or lactating participants, the healthcare providers (HCP) involved in their care or the care of their infants, if applicable, and Albireo pharmacovigilance.

Study start date is either start of data collection or first patient enrolled whatever occurs earlier.

The surveillance program is strictly observational; the schedule of office visits and all treatment regimens are determined by HCPs. Only data that are routinely documented in patients' medical records as part of usual care will be collected.

No additional laboratory tests or HCP assessments will be required as part of this surveillance program.

Study Overview

• Status: Recruiting
• Conditions: Pregnancy Related

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Ipsen Clinical Study Enquiries; Phone Number: See e mail; Email: clinical.trials@ipsen.com

Study Locations

• United States
  • North Carolina
    • Wilmington, North Carolina, United States, 28401
      • Recruiting
      • Virtual Research Coordination Center Odevixibat (BYLVAY) Pregnancy Surveillance Program

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

The main population will be individuals of any age who are exposed to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and/or at any time during lactation (up to 12 months of infant age or weaning, whichever comes first).

Description

Inclusion Criteria:

• Exposure to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and/or at any time during lactation (up to 12 months of infant age or weaning, whichever comes first).
• Informed consent or IRB-/EC-approved waiver of informed consent (not applicable if reported by Albireo PV according to usual pharmacovigilance practices)

Exclusion Criteria:

• Refusal to provide informed consent, if required

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prevalence rate of major congenital malformations (MCM) at birth	An abnormality of body structure or function that is present at birth; is of prenatal origin (i.e. birth defect); has significant medical, social or cosmetic consequences for the affected individual; and typically requires medical intervention.	At birth

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prevalence rate of minor congenital malformations at birth		At birth
Prevalence rate of molar or ectopic pregnancy	Molar pregnancy is defined as an abnormal pregnancy that happens when a sperm fertilizes an egg that does not contain any genetic material. Ectopic pregnancy is defined as a pregnancy that occurs outside of the uterine cavity, usually in one of the fallopian tubes.	Throughout pregnancy, an average of 9 months
Prevalence rate of fetal loss	A fetal loss that occurs for any reason at any time during pregnancy. This includes spontaneous abortion (SAB), stillbirth, elective or therapeutic abortion, fetal loss (type not specified).	Throughout pregnancy, an average of 9 months
Prevalence rate of live birth	The birth of a living fetus at ≥ 20 gestational weeks or, if gestational age is unknown, weighing ≥ 350g.	At birth
Prevalence rate of preterm birth		At birth
Prevalence rate of small for gestational age (SGA)	Birth weight <10th percentile for sex and gestational age using standard growth charts for full and preterm live-born infants.	At birth
Prevalence rate of neonatal death		Within the first 28 days of life
Prevalence rate of infant death		Throughout the first year of life
Prevalence rate of postnatal growth deficiency	Weight, length or head circumference in <10th percentile for sex and age using standard growth charts.	Throughout the first year of life
Prevalence rate of infant developmental delay	Failure to achieve the developmental milestones for chronological age, as defined by the Centers for Disease Control and Prevention (CDC)	Throughout the first year of life
Prevalence rate of infant hospitalization due to serious illness	Infant hospital visit due to a serious (i.e. results in significant disability, incapacity, or death; is life-threatening; requires inpatient or prolonged hospitalization; or is considered medically important)	Throughout the first year of life

Collaborators and Investigators

• Sponsor: Ipsen
• Investigators: Study Director: Ipsen Medical Director, Ipsen

Study record dates

Study Major Dates

• Study Start (Actual): September 28, 2023
• Primary Completion (Estimated): May 31, 2032
• Study Completion (Estimated): May 31, 2032

Study Registration Dates

• First Submitted: February 2, 2024
• First Submitted That Met QC Criteria: February 12, 2024
• First Posted (Actual): February 14, 2024

Study Record Updates

• Last Update Posted (Actual): May 28, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Pregnancy; Lactation; Rare disease; PFIC
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Pathological Conditions, Signs and Symptoms; Behavior; Feeding Behavior; Rare Diseases; Breast Feeding
• Other Study ID Numbers: A4250-017

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

• IPD Sharing Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
• IPD Sharing Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No