# 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)

> **NCT02736188** · PHASE3 · TERMINATED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 143 (actual)

## Conditions studied

- Hereditary Inclusion Body Myopathy
- Distal Myopathy With Rimmed Vacuoles
- Distal Myopathy, Nonaka Type
- GNE Myopathy
- Quadriceps Sparing Myopathy

## Interventions

- **DRUG:** Aceneuramic Acid Extended-Release Tablets

## Key facts

- **NCT ID:** NCT02736188
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2016-05-02
- **Primary completion:** 2018-01-10
- **Final completion:** 2018-01-10
- **Target enrollment:** 143 (ACTUAL)
- **Last updated:** 2023-03-24


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02736188

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02736188, "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)". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT02736188. Licensed CC0.

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