CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 59 enrolled
Drug / intervention
SA-ER 500 mg +1 moredrug
Likely dose
SA-ER 500 mgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01830972
NCT01830972Phase 2Completed
CompareCT.gov

An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Ultragenyx Pharmaceutical Inc·interventional·Posted Apr 12, 2013·Updated Apr 11, 2018

In Brief

A Phase 2 clinical trial evaluating SA-ER 500 mg and SA-IR 500 mg for GNE Myopathy and Hereditary Inclusion Body Myopathy (HIBM). Completed, enrolled 59 participants across 4 sites in 2 countries.

Detailed Summary

The 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 \[SA-ER\]).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM)
CountriesIsrael, United States
Collaborators--

Timeline

Phase 2CompletedFinished
20132014201520162017201820192020202120222023202420252026
First PostedApr 12, 2013
Enrollment StartJun 4, 2013
Primary CompletionFeb 14, 2017
TodayJul 2, 2026
Enrollment to primary: 3.7 yearsPosted 13.2 years ago

Interventions

SA-ER 500 mgdrug

oral tablets

SA-IR 500 mgdrug

oral capsules