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The purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.
Purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AEB1102 | Experimental | Each patient may receive AEB1102 administered IV for up to approximately 4 years. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AEB1102 | Drug | modified human arginase I |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-related adverse events | Incidence of treatment-related adverse events | up to 4 years |
| Measure | Description | Time Frame |
|---|---|---|
| Cmax Cmin | Cmax Cmin | up to 4 years |
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Inclusion Criteria:
Exclusion Criteria:
1. Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances
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| Name | Affiliation | Role |
|---|---|---|
| Cortney Caudill | Aeglea Biotherapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine | Stanford | California | 94305 | United States | ||
| University of Florida |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33325055 | Derived | Diaz GA, Schulze A, McNutt MC, Leao-Teles E, Merritt JL 2nd, Enns GM, Batzios S, Bannick A, Zori RT, Sloan LS, Potts SL, Bubb G, Quinn AG. Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency. J Inherit Metab Dis. 2021 Jul;44(4):847-856. doi: 10.1002/jimd.12343. Epub 2021 Jan 26. |
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| ID | Term |
|---|---|
| D020162 | Hyperargininemia |
| ID | Term |
|---|---|
| D056806 | Urea Cycle Disorders, Inborn |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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| Gainesville |
| Florida |
| 32610 |
| United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| UTSW | Dallas | Texas | 75390 | United States |
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Centro Hospitalar S. Joao | Porto | Portugal |
| Great Ormond Street Hospital | London | United Kingdom |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |