Clinical trial NCT05536011: WAKIX® (Pitolisant) Pregnancy Registry
Official title: WAKIX® (Pitolisant) Pregnancy Registry: An Observational Study of the Safety of Pitolisant Exposure in Pregnant Women and Their Offspring
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pregnancy Related
Narcolepsy
Lead sponsor: Harmony Biosciences Management, Inc.
Sponsor class: INDUSTRY
First posted: 2022-09-10
Enrollment: 1329
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The narcolepsy study population will include three cohorts (cohort 1, 2 and 3) of pregnant women with a diagnosis of narcolepsy:

* Cohort 1, exposed to pitolisant at any time during pregnancy
* Cohort 2, unexposed to pitolisant or any comparator product at any time during pregnancy
* Cohort 3, unexposed to pitolisant but exposed to a comparator product at any time during pregnancy

The non-narcolepsy study population will include two cohorts (cohorts 4 and 5) of pregnant women without a diagnosis of narcolepsy:

* Cohort 4, exposed to pitolisant at any time during pregnancy
* Cohort 5, unexposed to pitolisant but exposed to a comparator product at any time during pregnancy

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.

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.

Eligibility 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)

Primary outcomes:
measure: Major congenital malformation
timeFrame: Enrollment through the infant's first year of life
Secondary outcomes:
measure: Minor congenital malformation
timeFrame: Enrollment through the infant's first year of life
measure: Pre-eclampsia
timeFrame: Enrollment through pregnancy outcome
measure: Eclampsia
timeFrame: Enrollment through pregnancy outcome
measure: Spontaneous abortion
timeFrame: Enrollment through pregnancy outcome
measure: Stillbirth
timeFrame: Enrollment through pregnancy outcome
measure: Elective termination
timeFrame: Enrollment through pregnancy outcome
measure: Preterm birth
timeFrame: Enrollment through the infant's first year of life
measure: Small for gestational age
timeFrame: Enrollment through the infant's first year of life
measure: Postnatal growth deficiency
timeFrame: Enrollment through the infant's first year of life
measure: Infant development deficiency
timeFrame: Enrollment through the infant's first year of life
Other outcomes:
Locations:
city: Morrisville
country: United States
facility: Evidera (PPD)
state: North Carolina
Study officials:
References: