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Sialic Acid Extended Release

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Ensayo de Ultragenyx Pharmaceutical Inc. en Hereditary Inclusion Body Myopathy (HIBM) (del registro, en inglés).

Fase

Fase 1

Estado

Completado

Participantes

26

previstos

Centros

2

Fin del objetivo primario

abr 2012

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en jul 2011.

Qué mide

Evaluate the safety of repeated doses of Sialic Acid - Extended Release (SA-ER) tablets in patients with HIBM (Each patient may participate approximately 4-6 weeks total, including 2 single dose (fast/fed) treatment periods followed by a 7-day repeat treatment period.) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroHereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients. The effective replacement of the missing SA substrate is theoretically simple, and, in animal models, replacement with SA showed significant restoration of sialylation biochemistry and excellent reduction in muscle disease. These data show that replacement can achieve significant clinical benefit in muscle pathology, function, and survival.

Más ensayos de RARE

Todos los de RARE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01359319), actualizada en may 2012.