Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Summary

The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.

Timeline

Start

2016-05-02

Primary completion

2018-01-10

Completion

2018-01-10

Outcome

Missed primary endpoint

registry analysis (superiority test); Ace-ER 6 g/Day (Parent Study Treatment: Ace-ER 6 g/Day) vs Ace-ER 6 g/Day (Parent Study Treatment: Placebo); p = 0.4287; LS Mean Difference 0.80 (95% CI -1.17 to 2.77); NCT02736188 ↗

release “a Phase 3 study evaluating aceneuramic acid extended release (Ace-ER) in patients with GNE Myopathy (GNEM) did not achieve its primary endpoint” ultragenyx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sialic Acid	Small molecule	2 g	Oral

Indications