Calendario›SNY›A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
Ensayo de Sanofi en Sphingomyelin Lipidosis (del registro, en inglés).
- Fase
- Sin fase asignada
- Estado
- Completado
- Participantes
- 84
- Centros
- 28
- Fin del objetivo primario
- may 2023
previstos
fecha real
Qué significa cada fase y cada estado.
Estudio de intervención, de un solo grupo, abierto. Comenzó en sep 2019.
Qué mide
Time of first occurrence and recurrence of the clinical features and medical interventions related to chronic ASMD (Minimum 2 years) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroPrimary Objective: * To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD * To describe Clinician-Reported Outcomes (ClinROs) and Patient-Reported Outcomes (PROs) at enrollment and their evolution over time; disease severity at the time of diagnosis and its evolution over time Secondary Objectives: * To describe abnormal values in laboratory parameters and all values of specific clinical and imaging assessments at the time of diagnosis and their evolution over time * To study the use and applicability towards validation of a newly developed ASMD disease severity scoring system * To study the use and applicability towards validation of a newly developed ASMD PRO tool * To describe ASMD-related disease burden among patients with ASMD, caregivers, and healthcare resource utilization * To describe the association between patient demographics (eg, age, gender, race, Ashkenazi ancestry) and genotype with selected clinical features in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD
Más ensayos de SNY
| Ensayo | Fase | Estado | Lectura de resultados |
|---|---|---|---|
| Avalglucosidase alfa · Mini-COMET | Fase 2 | En marcha, sin reclutar | sep 2019 (real) |
| Yellow fever vaccine | Fase 2 | En marcha, sin reclutar | may 2022 (real) |
| Yellow fever vaccine · VYF02 | Fase 2 | En marcha, sin reclutar | jun 2022 (real) |
| SAR441344 IV | Fase 2 | En marcha, sin reclutar | sep 2022 (real) |
| Meningococcal Polysaccharide | Fase 3 | En marcha, sin reclutar | mar 2023 (real) |
| Isatuximab IV · IRAKLIA | Fase 3 | En marcha, sin reclutar | nov 2024 (real) |
Todos los de SNY, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT04106544), actualizada en ago 2023.