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SA-ER 500 mg

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

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

Más ensayos de: Miopatía por GNE.

Fase

Fase 2

Estado

Completado

Participantes

59

previstos

Centros

4

Fin del objetivo primario

feb 2017

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en jun 2013.

Qué mide

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation (From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per day) in the treatment of participants with GNE myopathy (Part II) over a 6 month treatment period; evaluate the safety of SA treatment at both 6g/day and 12 g/day (Part III \[SA-ER/SA-IR\] and Part IV \[SA-ER\]).

Más ensayos de RARE

Todos los de RARE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01830972), actualizada en mar 2018.