Extension Study for Phase 2 of Sialic Acid Tablet Announced Not Yet Recruiting

It's official that Ultragenyx will do  an extended study using Sialic Acid Extended Realease starting in June 2013 for the next 3 years. They plan to accept 45 patients.  Please refer to the link below.  Also, the National Institutes are still doing study on Natural History of patients with HIBM and they anre in Phase 1 trial of ManNac.  I would urge patients with HIBM to contact them.

 is not yet open for participant recruitment.

Verified April 2013 by Ultragenyx Pharmaceutical Inc

Sponsor:

Ultragenyx Pharmaceutical Inc

Information provided by (Responsible Party):

Ultragenyx Pharmaceutical Inc

ClinicalTrials.gov Identifier:

NCT01830972

First received: April 10, 2013

Last updated: NA

Last verified: April 2013

History: No changes posted

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  Purpose

GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid(SA). The purpose of the study is to measure long term safety and the effects of Sialic Acid-Extended Release (SA-ER) pills.

Condition	Intervention	Phase
GNE Myopathy HIBM	Drug: SA-ER tablets	Phase 2

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Resource links provided by NLM:

Genetics Home Reference related topics: inclusion body myopathy 2

MedlinePlus related topics: Muscle Disorders

U.S. FDA Resources 

Further study details as provided by Ultragenyx Pharmaceutical Inc:

Primary Outcome Measures:

• Assess long-term safety of 6000 mg/day SA-ER in HIBM subjects [ Time Frame: approximately 3 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	45
Study Start Date:	June 2013
Estimated Primary Completion Date:	June 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: open label, 6000 mg/day	Drug: SA-ER tablets

http://clinicaltrials.gov/ct2/show/NCT01830972?term=hibm&rank=5