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| ID | Type | Description | Link |
|---|---|---|---|
| U54NS115198 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Neurological Disorders and Stroke (NINDS) | NIH |
| Rare Diseases Clinical Research Network | NETWORK |
| Children's Hospital of Philadelphia | OTHER |
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This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-CDG
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AVTX-801, then Placebo | Experimental | Each treatment period is 24 weeks, with 6-week washout period in between. |
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| Placebo, then AVTX-801 | Placebo Comparator | Each treatment period is 24 weeks, with 6-week washout period in between. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AVTX-801 | Drug | Medical grade D-galactose dosage:2.0 g/kg/day |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of major motor seizure frequency | Seizure burden assessed by change from baseline in 28-day major motor seizure frequency | 28 days |
| Number of vomiting episodes | Vomiting frequency in 28-day from baseline | 28 days |
| Bristol Stool Form Scale (BSFS) | Constipation/diarrhea assessed by scale improvement toward normal stool forms from baseline in average daily 28-day Bristol Stool form scale. The BSFS classifies stool into 7 groups, type 1 (hardest) to type 7 (softest). Type 3 and Type 4 would be more indicative of normal stool. | average daily 28 days |
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Inclusion Criteria
Exclusion Criteria
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Mary Freeman, MS, CGC | Contact | 212-659-1434 | mary.freeman@mssm.edu |
| Name | Affiliation | Role |
|---|---|---|
| Eva Morava-Kozicz, MD, PhD | Icahn School of Medicine at Mount Sinai | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic Minnesota | Rochester | Minnesota | 55905 | United States |
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| ID | Term |
|---|---|
| D005690 | Galactose |
| ID | Term |
|---|---|
| D006601 | Hexoses |
| D009005 | Monosaccharides |
| D000073893 | Sugars |
| D002241 | Carbohydrates |
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| Placebo | Drug | Matching placebo |
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