WAKIX® (Pitolisant) Pregnancy Registry

Study Overview
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Brief Summary
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The WAKIX (pitolisant) Pregnancy Registry is a US-based, prospective, observational cohort study designed to evaluate the association between pitolisant exposure during pregnancy and subsequent maternal, fetal, and infant outcomes.

Detailed Description
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The WAKIX (pitolisant) Pregnancy Registry is a US-based, prospective, observational cohort study designed to evaluate the association between pitolisant exposure during pregnancy and subsequent maternal, fetal, and infant outcomes. Participation in the registry is voluntary and participants can withdraw their consent to participate at any time. Data will be collected from enrolled pregnant women and the healthcare providers (HCPs) involved in their care or the care of their infants, if applicable. The registry is strictly observational; the schedule of office visits and all treatment regimens will be determined by HCPs. Only data that are routinely documented in patients' medical records during the course of usual care will be collected. No additional laboratory tests or HCP assessments will be required as part of this registry. The design of this pregnancy registry follows current FDA guidance for designing and implementing pregnancy exposure registries.

Official Title
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WAKIX® (Pitolisant) Pregnancy Registry: An Observational Study of the Safety of Pitolisant Exposure in Pregnant Women and Their Offspring

Conditions
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Pregnancy Related, Narcolepsy

Intervention / Treatment
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* Drug: Pitolisant
* Drug: Comparator Products


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pregnant woman of any age Consent to participate Authorization for her HCP(s) to provide data to the registry For participants with a diagnosis of narcolepsy: exposure to at least one dose of pitolisant or one dose of a comparator product, or, unexposed to pitolisant or comparator products at any time during pregnancy (i.e., any pregnant woman with a diagnosis of narcolepsy) For participants without a diagnosis of narcolepsy: exposure to at least one dose of pitolisant or one dose of a comparator product at any time during pregnancy Exclusion Criterion: • Occurrence of pregnancy outcome prior to first contact with the Registry Coordination Center (RCC) (i.e., retrospectively enrolled)
Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pitolisant-exposed participants with narcolepsy<br>Pregnant women with a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy | Drug: Pitolisant<br>* Exposure to at least one dose of pitolisant at any time during pregnancy<br>* Other names: Wakix;|
| Unexposed participants with narcolepsy<br>Pregnant women with a diagnosis of narcolepsy who are not exposed to pitolisant or a comparator product at any time during pregnancy | |
| Comparator-exposed participants with narcolepsy<br>Pregnant women with a diagnosis of narcolepsy who are not exposed to pitolisant at any time during pregnancy but who are exposed to comparator products at any time during pregnancy | Drug: Comparator Products<br>* Exposure to at least one dose of a comparator product at any time during pregnancy<br>* Other names: Amphetamines;|
| Pitolisant-exposed participants without narcolepsy<br>Pregnant women without a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy | Drug: Pitolisant<br>* Exposure to at least one dose of pitolisant at any time during pregnancy<br>* Other names: Wakix;|
| Comparator-exposed participants without narcolepsy<br>Pregnant women without a diagnosis of narcolepsy who are not exposed to pitolisant at any time during pregnancy but who are exposed to a comparator product at any time during pregnancy | Drug: Comparator Products<br>* Exposure to at least one dose of a comparator product at any time during pregnancy<br>* Other names: Amphetamines;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Major congenital malformation | 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. | Enrollment through the infant's first year of life | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Minor congenital malformation | 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. | Enrollment through the infant's first year of life | 
| Pre-eclampsia | High blood pressure and signs of liver or kidney damage occurring at >20 gestational weeks. | Enrollment through pregnancy outcome | 
| Eclampsia | Seizures or coma in a pregnant woman with pre-eclampsia. | Enrollment through pregnancy outcome | 
| Spontaneous abortion | An involuntary fetal loss or the expulsion of the products of conception occurring at <20 gestational weeks. | Enrollment through pregnancy outcome | 
| Stillbirth | As defined by the American College of Obstetricians and Gynecologists (ACOG), an involuntary fetal loss occurring at ≥20 gestational weeks or, if gestational age is unknown, a fetus weighing ≥350 g. | Enrollment through pregnancy outcome | 
| Elective termination | A voluntary fetal loss or interruption of pregnancy, including pregnancy termination that occurs electively, to preserve maternal health or due to fetal abnormalities. | Enrollment through pregnancy outcome | 
| Preterm birth | A live birth occurring at <37 gestational weeks. | Enrollment through the infant's first year of life | 
| Small for gestational age | Weight, length, or head circumference at birth in <10th percentile for sex and gestational age using standard growth charts for full and preterm live born infants. | Enrollment through the infant's first year of life | 
| Postnatal growth deficiency | Weight, length, or head circumference in <10th percentile for sex and chronological age using standard growth charts | Enrollment through the infant's first year of life | 
| Infant development deficiency | Failure to achieve the developmental milestones for chronological age, as defined by the Centers for Disease Control and Prevention (CDC). | Enrollment through the infant's first year of life |