Pregnancy Related
Conditions
Keywords
Rare disease, Primary Biliary Cholangitis, Pregnancy
Brief summary
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period \[LMP\]). Information will be collected from participants, their healthcare providers, published studies, and safety databases. Reports of pregnancy linked to elafibranor from clinical trials, spontaneous reports, or literature will also be included, with steps taken to avoid duplicates. The study begins once the first participant is enrolled and ends after the last mother and child data are collected. It is planned to run for about 10 years, with infant follow-up lasting up to 2 years, for a maximum total duration of 12 years and 9 months. The program is strictly observational. All medical care, visit schedules, and treatment decisions remain with healthcare providers. Only routine medical record data will be collected, and no extra tests or procedures are required. Participation is voluntary, and written informed consent will be obtained before enrollment.
Interventions
None listed
Sponsors
Ipsen
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Inclusion criteria
* Maternal exposure to at least one dose of elafibranor, either: Within three weeks prior to conception (based on the estimated date of LMP) or At any time during pregnancy (from the estimated date of conception through pregnancy outcome). * Participants who were previously enrolled in this program during a past pregnancy and who meet inclusion criteria #1 for the subsequent pregnancy are eligible to enroll again. * Informed consent or IRB/IEC-approved waiver of informed consent (not applicable if reported by Ipsen PV according to usual pharmacovigilance practices).
Exclusion criteria
* Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Prevalence of congenital malformations at birth | At birth |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of infant healthcare requirements and interventions not considered standard | Throughout the two years of life | including but not limited to: * Infant hospitalization due to serious illness * Emergency department visits * Specialist or other healthcare professional consultations * Developmental assessments and interventions * Therapeutic services * Educational support and adaptations |
| Incidence and nature of all adverse events in pregnant participants | Throughout pregnancy, an average of 9 months | — |
| Changes from baseline in Alkaline phosphatase (ALP) | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Bilirubin | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Alanine aminotransferase (ALT) | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Aspartate aminotransferase (AST) | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Gamma-glutamyl transferase (GGT) | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Albumin | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baselinein Bile Acids | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Creatine phosphokinase (CPK) | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | — |
| Changes from baseline in Lipid Profile | From the time of first elafibranor exposure throughout pregnancy and postpartum, an average of 2 years and 9 months | Lipid profile includes total cholesterol, low density lipoproteins (LDL-C), high density lipoprotein (HDL-C), very low density lipoprotein and triglycerides |
| Prevalence infant death | Throughout the first year of life | — |
| Prevalence of postnatal growth deficiency | Throughout the two years of life | Weight, length, or head circumference in \<10th percentile for sex and age using standard growth charts |
| Prevalence of major congenital malformations at birth | 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 |
| Prevalence of minor congenital malformations at birth | At birth | An anomaly or abnormality of body structure that is present at birth, is of prenatal origin (i.e. birth defect), poses no significant health problem in the neonatal period, and tends to have limited social or cosmetic consequences for the affected individual |
| Prevalence of molar/ectopic pregnancy | Throughout pregnancy, an average of 9 months | 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. |
| Prevalence of fetal loss | Throughout pregnancy, an average of 9 months | 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). |
| Prevalence of live birth | At birth | The birth of a living fetus at ≥ 20 gestational weeks or, if gestational age is unknown, weighing ≥ 350g. |
| Prevalence rate of premature delivery | At birth | A live birth occurring at \<37 gestational weeks. |
| Prevalence of infants born small for gestational age (SGA) at birth | At birth | Birth weight \<10th percentile for sex and gestational age using standard growth charts for full and preterm live-born infants. |
| Prevalence of infant developmental delay | Throughout the two years of life | Failure to achieve the developmental milestones for chronological age, as defined by the Centers for Disease Control and Prevention (CDC) |
| Prevalence of neonatal death | Within the first 28 days of life | — |
Countries
United States
Contacts
CONTACTIpsen Clinical Study Enquiries
STUDY_DIRECTORIpsen Medical Director
Ipsen
Outcome results
None listed