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Also known as Sialic Acid Extended Release (SA-ER) Tablets, Sialic Acid Extended Release (SA-ER)
Sialic Acid · 2 trials · 3 indications
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.
Safety will be evaluated in terms of the incidence and frequency of treatment-emergent adverse events (TEAEs), including clinically significant changes from baseline to scheduled timepoints in clinical laboratory values, vital signs, or physical and neurologic examination findings. Medical history and reported clinical symptoms, including increasing muscle weakness, fatigue, or pain.
| 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). |
| 650 mg (single dose only) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 1,950 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 2,925 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 4,875 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 6,000 mg (single and repeat dose) | EXPERIMENTAL | Each patient will be sequentially assigned to a specific dose level and will receive two single-dose exposures at that same dose level (fasted and fed). The low-dose cohorts will be filled before assigning higher-dose cohorts. The patient will then be assigned to receive one repeat-dose regimen. The lower-dose repeat-dose cohorts will be filled before proceeding to higher repeat-dose levels. |
| 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). |
| Sialic Acid Extended Release (SA-ER) Tablets | DRUG | Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis. |
| Sialic Acid Extended Release (SA-ER) Tables | DRUG | PPatients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis. |
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 |
Sialic acid is being developed as an oral extended-release tablet treatment for GNE myopathy, also called hereditary inclusion body myopathy (HIBM). Ultragenyx Pharmaceutical Inc. is studying it as a substrate replacement approach for this rare inherited muscle disorder. It is investigational and has not been approved for any indication.
Sialic acid extended release (SA-ER) tablets are being developed by Ultragenyx Pharmaceutical Inc., which trades on the Nasdaq under the ticker RARE. The company sponsored the clinical program evaluating the drug in patients with GNE myopathy or hereditary inclusion body myopathy.
Sialic acid extended release tablets have completed Phase 2 development for GNE myopathy and hereditary inclusion body myopathy. The Phase 2 trial, NCT01517880, has finished, and no trials of this program are currently active. The drug remains investigational and is not FDA approved.
Sialic acid extended release tablets were studied in two completed trials. NCT01517880 was a Phase 2 randomized, double-blind, placebo-controlled study of 46 patients with GNE myopathy or hereditary inclusion body myopathy in the United States and Israel. NCT01359319 was a Phase 1 safety and pharmacokinetics study of 26 patients with hereditary inclusion body myopathy in the United States.
Yes. Sialic acid and sialic acid extended release (SA-ER) tablets refer to the same Ultragenyx investigational product for GNE myopathy and hereditary inclusion body myopathy. The extended-release tablet formulation is the version tested in the clinical program, including the Phase 2 trial NCT01517880.
Sialic acid extended release is a small molecule therapeutic being developed for GNE myopathy and hereditary inclusion body myopathy. It is formulated as an oral extended-release tablet. Ultragenyx Pharmaceutical Inc. evaluated it in a placebo-controlled Phase 2 trial that enrolled 46 patients, and the program is investigational.