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Mucopolysaccharidosis Type II Natural History

Ensayo de REGENXBIO Inc. en Mucopolisacaridosis II.

Más ensayos de: Mucopolisacaridosis II.

Fase

no aplica

Estado

Completado

Participantes

36

previstos

Centros

3

Fin del objetivo primario

mar 2022

fecha real

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en sep 2017.

Qué mide

Cognitive function over time, as indicated by results of neurocognitive measures documented in medical chart. (Up to 10 years old) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroMucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in characteristic storage lesions and diverse disease sequelae, and in patients with the more severe form of the disease, irreversible neurocognitive decline and higher morbidity and mortality than in patients with the attenuated form of the disease. There is currently limited information on the natural history of MPS II, especially with respect to neurocognitive decline in patients with the more severe form of the disease. This study is planned to be an observational medical records review study (data collected retrospectively and no investigational product treatment or procedures) in subjects with the severe form of MPS II. Collectively, the data may inform the design of future MPS II gene therapy treatment studies and may be utilized as historical comparative control data.

Más ensayos de RGNX

Todos los de RGNX, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT03529786), actualizada en abr 2022.