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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | PHASE2 | COMPLETED | 46 | — | — | May 1, 2012 | Nov 1, 2013 | Jun 16, 2016 | 4 | United States, Israel |
To evaluate the effect of SA-ER treatment on improvement of biochemical measures of sialylation and pathology in muscle. On mobility, strength, and function using a series of quantitative and physical performance measures and quality of life using patient-reported outcome measures.
| Arm | Type | Description |
|---|---|---|
| 6,000 mg SA-ER | EXPERIMENTAL | Subjects will receive this dose for the duration of the study (total study duration of 48 weeks). |
| Placebo | PLACEBO_COMPARATOR | Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (48 weeks total study duration). |
| 3,000 mg SA-ER | EXPERIMENTAL | Subjects will receive this dose for the duration of the study (total study duration of 48 weeks). |
| Name | Type | Description |
|---|---|---|
| Sialic Acid Extended Release (SA-ER) | DRUG | SA-ER will be administered in doses of 3000mg per day or 6000mg per day |
| Placebo | DRUG | Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (total study duration 48 weeks). |
Inclusion Criteria: 1. Must be between 18 and 65 years of age, inclusive. 2. Must be willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures. 3. Must have a documented diagnosis of GNE myopathy, HIBM...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |