WAKIX® (Pitolisant) Pregnancy Registry
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Pitolisant-exposed participants with narcolepsy Pregnant women with a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy |
Drug: Pitolisant
Exposure to at least one dose of pitolisant at any time during pregnancy
Other Names:
|
| Unexposed participants with narcolepsy 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 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
Exposure to at least one dose of a comparator product at any time during pregnancy
Other Names:
|
| Pitolisant-exposed participants without narcolepsy Pregnant women without a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy |
Drug: Pitolisant
Exposure to at least one dose of pitolisant at any time during pregnancy
Other Names:
|
| Comparator-exposed participants without narcolepsy 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
Exposure to at least one dose of a comparator product at any time during pregnancy
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Major congenital malformation [Enrollment through the infant's first year of life]
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.
Secondary Outcome Measures
- Minor congenital malformation [Enrollment through the infant's first year of life]
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.
- Pre-eclampsia [Enrollment through pregnancy outcome]
High blood pressure and signs of liver or kidney damage occurring at >20 gestational weeks.
- Eclampsia [Enrollment through pregnancy outcome]
Seizures or coma in a pregnant woman with pre-eclampsia.
- Spontaneous abortion [Enrollment through pregnancy outcome]
An involuntary fetal loss or the expulsion of the products of conception occurring at <20 gestational weeks.
- Stillbirth [Enrollment through pregnancy outcome]
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.
- Elective termination [Enrollment through pregnancy outcome]
A voluntary fetal loss or interruption of pregnancy, including pregnancy termination that occurs electively, to preserve maternal health or due to fetal abnormalities.
- Preterm birth [Enrollment through the infant's first year of life]
A live birth occurring at <37 gestational weeks.
- Small for gestational age [Enrollment through the infant's first year of life]
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.
- Postnatal growth deficiency [Enrollment through the infant's first year of life]
Weight, length, or head circumference in <10th percentile for sex and chronological age using standard growth charts
- Infant development deficiency [Enrollment through the infant's first year of life]
Failure to achieve the developmental milestones for chronological age, as defined by the Centers for Disease Control and Prevention (CDC).
Eligibility Criteria
Criteria
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)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Evidera (PPD) | Morrisville | North Carolina | United States | 28403 |
Sponsors and Collaborators
- Harmony Biosciences, LLC
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HBS-101-CL-008