WAKIX® (Pitolisant) Pregnancy Registry

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

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.

Study Overview

• Status: Recruiting
• Conditions: Pregnancy Related; Narcolepsy
• Intervention / Treatment: Drug: Pitolisant; Drug: Comparator Products

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 Type: Observational
• Enrollment (Estimated): 1329

Contacts and Locations

• Study Contact: Name: WAKIX Pregnancy Registry Associate; Phone Number: 1-877-302-2813; Email: wakixpregnancyregistry@ppd.com

Study Locations

• United States
  • North Carolina
    • Morrisville, North Carolina, United States, 28403
      • Recruiting
      • Evidera (PPD)
      • Contact: Kristin Veley, PharmD, MPH

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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

Description

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)

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

5

Cohorts and Interventions

Group / Cohort	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: Wakix
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: Methylphenidate; Modafinil/armodafinil; Sodium oxybate; Oxybate mixed salts; Solriamfetol; Amphetamines
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: Wakix
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: Methylphenidate; Modafinil/armodafinil; Sodium oxybate; Oxybate mixed salts; Solriamfetol; Amphetamines

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Harmony Biosciences Management, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 24, 2021
• Primary Completion (Estimated): June 30, 2030
• Study Completion (Estimated): June 30, 2030

Study Registration Dates

• First Submitted: September 7, 2022
• First Submitted That Met QC Criteria: September 7, 2022
• First Posted (Actual): September 10, 2022

Study Record Updates

• Last Update Posted (Actual): August 5, 2025
• Last Update Submitted That Met QC Criteria: July 31, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Mental Disorders; Sleep Wake Disorders; Sleep Disorders, Intrinsic; Dyssomnias; Disorders of Excessive Somnolence; Narcolepsy; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Autonomic Agents; Peripheral Nervous System Agents; Anesthetics; Central Nervous System Depressants; Neurotransmitter Agents; Membrane Transport Modulators; Adjuvants, Anesthesia; Anesthetics, Intravenous; Anesthetics, General; Adrenergic Agents; Central Nervous System Stimulants; Dopamine Uptake Inhibitors; Neurotransmitter Uptake Inhibitors; Dopamine Agents; Adrenergic Uptake Inhibitors; Sympathomimetics; Cytochrome P-450 Enzyme Inducers; Cytochrome P-450 CYP3A Inducers; Wakefulness-Promoting Agents; Modafinil; Methylphenidate; Amphetamine; Sodium Oxybate
• Other Study ID Numbers: HBS-101-CL-008

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No