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).
Visão geral do estudo
Status
Intervenção / Tratamento
Descrição detalhada
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).
Tipo de estudo
Inscrição (Estimado)
Contactos e Locais
Contato de estudo
- Nome: Marie Benhammani-Godard
- Número de telefone: 0033158411190
- E-mail: marie.godard@aphp.fr
Estude backup de contato
- Nome: Alexandre ROUEN
- Número de telefone: +33 (0)1 42 64 86 26
- E-mail: alexandre.rouen@aphp.fr
Locais de estudo
-
-
IDF
-
Paris, IDF, França, 75014
- Sleep and Vigilance Center, Hôtel-Dieu
-
-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
Patients diagnosed with central hypersomnia and diagnosed and/or followed-up at our site.
Descrição
Inclusion Criteria:
- Patients who have had at least one consultation or examination at the Sleep and Vigilance Center at Hôtel-Dieu since January 1, 2005;
- Patients diagnosed with narcolepsy (type 1 or type 2) or idiopathic hypersomnia;
- Adult patients (aged 18 or over);
- Patients who have not objected to the processing of their data.
Exclusion Criteria:
- Minors;
- Patients who object to the processing of their data;
- Patients under guardianship or conservatorship.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Modelos de observação: COHORT
- Perspectivas de Tempo: OTHER
Coortes e Intervenções
Grupo / Coorte |
Intervenção / Tratamento |
|---|---|
|
Central hypersomnia
Patients diagnosed with central hypersomnia |
Non applicable |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
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.
Prazo: 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
|
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
Prazo: 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: 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
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Annual percentage of patients taking at least two treatments for their central hypersomnia.
Prazo: 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.
|
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.
Prazo: During first analyses
|
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.
Prazo: During first analyses
|
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.
Prazo: 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 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.
Prazo: 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. |
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
Prazo: 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.
|
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
Prazo: 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.
|
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
Prazo: 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 |
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
Prazo: 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 |
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.
Prazo: 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. |
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
Prazo: 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 |
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
Prazo: 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 (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.
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Alexandre ROUEN, APHP
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- APHP251690
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .