[Translation] A single-arm, open-label, multicenter clinical study to evaluate the safety, efficacy, and pharmacokinetics of carboglutamate dispersible tablets in the treatment of hyperammonemia caused by N-acetylglutamate synthetase deficiency and organic acidemia (propionic acidemia, methylmalonic acidemia, or isovaleric acidemia)
1. 高氨血症中国患者中评价卡谷氨酸分散片的AE发生率和严重程度。
2. 评价卡谷氨酸分散片在缓解高氨血症方面的有效性。
3. 评价卡谷氨酸分散片在高氨血症中国患者中的药代动力学特征并探索协变量(如年龄、体重、性别和其他)对卡谷氨酸PK的影响。
[Translation] 1. To evaluate the incidence and severity of AEs of carboglutamate dispersible tablets in Chinese patients with hyperammonemia.
2. To evaluate the effectiveness of carboglutamate dispersible tablets in relieving hyperammonemia.
3. To evaluate the pharmacokinetic characteristics of carboglutamate dispersible tablets in Chinese patients with hyperammonemia and explore the effects of covariates (such as age, weight, gender, and others) on carboglutamate PK.