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Also known as aceneuramic acid extended-release (Ace-ER)
aceneuramic acid · 1 trial · 4 indications
Muscle strength based on the maximum voluntary isometric contraction (MVIC) against a dynamometer was measured bilaterally in the following upper extremity muscle groups: gross grip, shoulder abductors, elbow flexors, and elbow extensors. The UEC is derived from the sum of the average of the right and left total force values (measured in kg).
| Arm | Type | Description |
|---|---|---|
| Aceneuramic Acid Extended-Release (Ace-ER) | EXPERIMENTAL | Ace-ER 6 g/day, divided 3 times per day (TID) for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo TID for 48 weeks. |
| Name | Type | Description |
|---|---|---|
| aceneuramic acid extended-release (Ace-ER) | DRUG | tablets for oral use |
| Placebo | DRUG | tablets for oral use |
Inclusion Criteria: * Male or female, aged 18 to 55 years, inclusive * Willing and able to provide written, signed informed consent after the nature of the study has been explained, and before any research-related procedures are conducted * Have a documented diagnosis of GNEM, HIBM, distal myopathy...
Aceneuramic acid is being developed for the treatment of Hereditary Inclusion Body Myopathy, a rare inherited muscle disorder. It is an investigational small molecule studied in a Phase 3 clinical program. The condition is also referred to as GNE myopathy, distal myopathy with rimmed vacuoles, and Nonaka type distal myopathy.
Aceneuramic acid is being developed by Ultragenyx Pharmaceutical Inc., which trades on the Nasdaq under the ticker RARE. The company is the sponsor of the Phase 3 clinical program evaluating the drug in patients with Hereditary Inclusion Body Myopathy and related GNE myopathy conditions.
Aceneuramic acid is in Phase 3 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 3 study has been completed, and no trials of this drug are currently listed as active.
Aceneuramic acid has been evaluated in one Phase 3 trial, NCT02377921, titled a Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNEM or Hereditary Inclusion Body Myopathy. The completed study enrolled 89 patients across the United States, Bulgaria, Canada, France, Israel, Italy, and the United Kingdom.
Yes. Aceneuramic acid is also known as aceneuramic acid extended-release, abbreviated Ace-ER. Both names refer to the same investigational small molecule being developed by Ultragenyx Pharmaceutical Inc. for Hereditary Inclusion Body Myopathy and related GNE myopathy conditions.
The Phase 3 trial NCT02377921 was a randomized, double-blind, placebo-controlled study. It enrolled 89 patients aged 18 years and older, with both sexes eligible and no healthy volunteers included. The study evaluated aceneuramic acid in Hereditary Inclusion Body Myopathy, GNE myopathy, and related distal myopathy conditions, and it has been completed.