# A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

> **NCT01517880** · PHASE2 · COMPLETED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 46 (actual)

## Conditions studied

- GNE Myopathy
- Hereditary Inclusion Body Myopathy

## Interventions

- **DRUG:** Sialic Acid Extended Release (SA-ER)
- **DRUG:** Placebo

## Key facts

- **NCT ID:** NCT01517880
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2012-05
- **Primary completion:** 2013-11
- **Final completion:** 2013-11
- **Target enrollment:** 46 (ACTUAL)
- **Last updated:** 2016-06-16


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01517880

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01517880, "A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT01517880. Licensed CC0.

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