# A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

> **NCT02731690** · PHASE2 · TERMINATED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 42 (actual)

## Conditions studied

- Hereditary Inclusion Body Myopathy
- Distal Myopathy With Rimmed Vacuoles
- Distal Myopathy, Nonaka Type
- GNE Myopathy
- Quadriceps Sparing Myopathy
- Inclusion Body Myopathy 2

## Interventions

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

## Key facts

- **NCT ID:** NCT02731690
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2016-04-29
- **Primary completion:** 2018-01-10
- **Final completion:** 2018-01-10
- **Target enrollment:** 42 (ACTUAL)
- **Last updated:** 2019-02-19


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02731690

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02731690, "A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment". Retrieved via AI Analytics 2026-07-07 from https://api.ai-analytics.org/clinical/NCT02731690. Licensed CC0.

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