Evolution of the Treatment of Rare Hypersomnias in a Reference Center Certified Since 2005 (EPHyRa)

Central hypersomnias are rare diseases that include narcolepsy and idiopathic hypersomnia.

The Sleep and Vigilance Center at Hôtel-Dieu has been certified as a Rare Disease Reference Center (CRMR) for these conditions since 2005.

Scientific advances and changes to the ICSD (International Classification of Sleep Disorders) since 2005 (diagnostic criteria and drugs brought to market) have greatly improved the care of patients with central hypersomnia.

This retrospective and prospective study aims to evaluate how each advance translates into concrete terms in the care of patients followed at our center.

Main objective :

Determine whether new treatments are prescribed to replace treatments already on the market (reduction in prescriptions for treatments previously marketed) or whether they enable patients who have reached a therapeutic impasse to be treated (no reduction in prescriptions for treatments previously marketed).

Study Overview

• Status: Not yet recruiting
• Conditions: Narcolepsy Type 1; Hypersomnia, Idiopathic; Narcolepsy and Hypersomnia; Narcolepsy Type 2
• Intervention / Treatment: Other: Non applicable

Detailed Description

Central hypersomnias are rare diseases that include narcolepsy and idiopathic hypersomnia.

The Sleep and Vigilance Center at Hôtel-Dieu has been certified as a Rare Disease Reference Center (CRMR) for these conditions since 2005.

Scientific advances and changes to the ICSD (International Classification of Sleep Disorders) since 2005 (diagnostic criteria and drugs brought to market) have greatly improved the care of patients with central hypersomnia.

This retrospective and prospective study aims to evaluate how each advance translates into concrete terms in the care of patients followed at our center.

Main objective :

Determine whether new treatments are prescribed to replace treatments already on the market (reduction in prescriptions for treatments previously marketed) or whether they enable patients who have reached a therapeutic impasse to be treated (no reduction in prescriptions for treatments previously marketed).

• Study Type: Observational
• Enrollment (Estimated): 1500

Contacts and Locations

• Study Contact: Name: Marie Benhammani-Godard; Phone Number: 0033158411190; Email: marie.godard@aphp.fr
• Study Contact Backup: Name: Alexandre ROUEN; Phone Number: +33 (0)1 42 64 86 26; Email: alexandre.rouen@aphp.fr

Study Locations

• France
  • IDF
    • Paris, IDF, France, 75014
      • Sleep and Vigilance Center, Hôtel-Dieu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with central hypersomnia and diagnosed and/or followed-up at our site.

Description

Inclusion Criteria:

1. Patients who have had at least one consultation or examination at the Sleep and Vigilance Center at Hôtel-Dieu since January 1, 2005;
2. Patients diagnosed with narcolepsy (type 1 or type 2) or idiopathic hypersomnia;
3. Adult patients (aged 18 or over);
4. Patients who have not objected to the processing of their data.

Exclusion Criteria:

1. Minors;
2. Patients who object to the processing of their data;
3. Patients under guardianship or conservatorship.

Study Plan

How is the study designed?

Design Details

• Observational Models: COHORT
• Time Perspectives: OTHER

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Central hypersomnia; Patients diagnosed with central hypersomnia	Other: Non applicable; Non applicable

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ratio of the number of patients who were prescribed this treatment during the year to the number of patients who had at least one consultation at the center during the same year.	Ratio of the number of patients who were prescribed this treatment during the year to the number of patients who had at least one consultation at the center during the same year. Annual change in prescriptions for each treatment	For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3
Number of days of treatment	Number of days of treatment: mean + standard deviation for patients who received this treatment during the year, median and quartiles. Annual change in prescriptions for each treatment	For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annual percentage of patients taking at least two treatments for their central hypersomnia.	Annual percentage of patients taking at least two treatments for their central hypersomnia.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Number of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.	Number of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.	During first analyses
Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.	Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.	During first analyses
Number of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.	Number of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Percentage of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.	Percentage of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Epworth scale	Reduction in sleepiness (Epworth scale and wakefulness maintenance tests) average for each treatment and combination of treatments.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Wakefulness maintenance tests	Reduction in sleepiness (Epworth scale and wakefulness maintenance tests) average for each treatment and combination of treatments.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Average age of these patients at the onset of cataplexy	Average age of these patients at the onset of cataplexy	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Average number of years between initial diagnosis and onset of cataplexy	Average number of years between initial diagnosis and onset of cataplexy	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Average number of years between first symptoms and onset of cataplexy.	Average number of years between first symptoms and onset of cataplexy.	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Adverse events collected during medical consultations	Adverse events collected during medical consultations	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
Effects on other components of the pathologies	Effects on other components of the pathologies (via IHSS scores, NSS, fatigue and brain fog scale, etc.).	This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: URC-CIC Paris Descartes Necker Cochin
• Investigators: Principal Investigator: Alexandre ROUEN, APHP

Study record dates

Study Major Dates

• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): November 1, 2036
• Study Completion (Estimated): November 1, 2036

Study Registration Dates

• First Submitted: November 5, 2026
• First Submitted That Met QC Criteria: November 5, 2026
• First Posted (Actual): November 12, 2026

Study Record Updates

• Last Update Posted (Actual): November 12, 2026
• Last Update Submitted That Met QC Criteria: November 5, 2026
• Last Verified: November 1, 2026

More Information

Terms related to this study

• Keywords: Idiopathic hypersomnia; Narcolepsy; Central hypersomnia
• Additional Relevant MeSH Terms: Nervous System Diseases; Mental Disorders; Sleep Wake Disorders; Sleep Disorders, Intrinsic; Dyssomnias; Disorders of Excessive Somnolence; Narcolepsy; Idiopathic Hypersomnia
• Other Study ID Numbers: APHP251690

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No