WAKIX® (Pitolisant) Pregnancy Registry

NCT05536011 | Recruiting FDA Drug

• 1 other identifier: HBS-101-CL-008
• Study Type: observational
• Target: 1,329
• Locations: 1 country
• Sites: 1

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.

Conditions

Pregnancy Related; Narcolepsy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• Minor congenital malformation

  Enrollment through the infant's first year of life

• Pre-eclampsia

  Enrollment through pregnancy outcome

• Eclampsia

  Enrollment through pregnancy outcome

• Spontaneous abortion

  Enrollment through pregnancy outcome

• Stillbirth

  Enrollment through pregnancy outcome

Study Arms (5)

Pitolisant-exposed participants with narcolepsy

Pregnant women with a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy

Drug: Pitolisant

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

Pitolisant-exposed participants without narcolepsy

Pregnant women without a diagnosis of narcolepsy who are exposed to pitolisant at any time during pregnancy

Drug: Pitolisant

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

Interventions

Pitolisant DRUG

Exposure to at least one dose of pitolisant at any time during pregnancy

Also known as: Wakix

Pitolisant-exposed participants with narcolepsy; Pitolisant-exposed participants without narcolepsy

Comparator Products DRUG

Exposure to at least one dose of a comparator product at any time during pregnancy

Also known as: Modafinil/armodafinil, Sodium oxybate, Oxybate mixed salts, Solriamfetol, Methylphenidate, Amphetamines

Comparator-exposed participants with narcolepsy; Comparator-exposed participants without narcolepsy

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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
• Occurrence of pregnancy outcome prior to first contact with the Registry Coordination Center (RCC) (i.e., retrospectively enrolled)

Sponsors & Collaborators

• Harmony Biosciences Management, Inc.: lead

Study Sites (1)

Evidera (PPD)

Morrisville, North Carolina, 28403, United States

RECRUITING

MeSH Terms

Conditions

Narcolepsy

Interventions

pitolisant; Modafinil; Sodium Oxybate; solriamfetol; Methylphenidate; Amphetamines

Condition Hierarchy (Ancestors)

Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Intervention Hierarchy (Ancestors)

Benzhydryl Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Hydroxybutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Hydroxy Acids; Phenylacetates; Acids, Carbocyclic; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Phenethylamines; Ethylamines; Amines

Central Study Contacts

WAKIX Pregnancy Registry Associate

CONTACT

1-877-302-2813; wakixpregnancyregistry@ppd.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 21 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2022
• First Posted: September 10, 2022
• Study Start: August 24, 2021
• Primary Completion (Estimated): June 30, 2030
• Study Completion (Estimated): June 30, 2030
• Last Updated: August 5, 2025

Record last verified: 2025-07

Locations

US (1)