From: Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan
Preferred term | Trial-1a | Trial-2 | ||||
---|---|---|---|---|---|---|
800 mg × 1 n = 3 | 800 mg × 3 n = 3 | 800 mg × 3 × 5 n = 3 | 2,000 mg × 1 n = 3 | 2,000 mg × 3  n = 3 | 2,000 mg × 3 × 7 n = 3 | |
Subjects with any AE, n (%) | 3 (100) | 3 (100) | 3 (100) | 1 (33.3) | 2 (66.7) | 2 (66.7) |
The number of AEs, n | 14 | 17 | 22 | 1 | 3 | 2 |
Blood albumin decreased | 1 (33.3) | 2 (66.7) | 3 (100) | |||
Protein total decreased | 2 (66.7) | 1 (33.3) | 2 (66.7) | |||
Haematcrit decreased | 1 (33.3) | 2 (66.7) | 1 (33.3) | |||
Protein urine present | 2 (66.7) | 1 (33.3) | ||||
Albumin globulin ratio decreased | 2 (66.7) | 1 (33.3) | ||||
Platelet count decreased | 1 (33.3) | 1 (33.3) | ||||
Blood cholinesterase Increased | 1 (33.3) | 1 (33.3) | ||||
Blood phosphorus increased | 1 (33.3) | 1 (33.3) | ||||
Blood urea decreased | 1 (33.3) | 1 (33.3) | ||||
Specific gravity urine Increased | 1 (33.3) | 1 (33.3) | ||||
Blood potassium decreased | 1 (33.3) | 1 (33.3) | ||||
Red blood cell count decreased | 1 (33.3) | 1 (33.3) | ||||
Blood urine present | 1 (33.3) | 1 (33.3) | ||||
eczema | 1 (33.3) | |||||
Blood triglycerides increased | 2 (66.7) | 1 (33.3) | ||||
Blood lactate dehydrogenase increased | 1 (33.3) | |||||
Fall | 1 (33.3) |