Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02377921 | Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) | PHASE3 | COMPLETED | 89 | — | — | May 20, 2015 | Jun 9, 2017 | Jun 27, 2019 | 13 | United States, Bulgaria +5 |
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...