GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3. This compound was researched and developed as a drug for treatment of Fabry disease. This study, MGM115806, will be the first administration of GR181413A/AT1001 to Japanese subjects to investigate the safety, tolerability and pharmacokinetics of single oral doses in healthy Japanese adult subjects. Approximately 12 subjects will receive three treatments of 50, 150 and 450 mg GR181413A/AT1001 under fasted conditions plus placebo in a dose ascending crossover design. Serial pharmacokinetic samples will be collected and safety assessments will be performed following each dose. The pharmacokinetics and dose proportionality of GR181413A/AT1001 after single oral doses of GR181413A/AT1001 at the dose levels of 50, 150 and 450 mg under fasted conditions will be assessed.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Masking
SINGLE
Enrollment
14
Powder for reconstitution
Size 2, hard gelatin capsule, white opaque / blue opaque
Matched, Size 2, hard gelatin capsule, white opaque/blue opaque
Solution matched
GSK Investigational Site
Randwick, New South Wales, Australia
Profile of plasma pharmacokinetics
AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F
Time frame: 0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose
Number of Participants with adverse events
Time frame: up to 24 hr
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
Time frame: 0, 24h post dose
Change from baseline in vital signs (blood pressure and heart rate)
Time frame: 0 ,1 , 2, 3, 4, 5, 6, 24h post dose
Change from baseline in 12-lead ECG
Time frame: 0, i, 2, 3, 6, 24h post dose
Profile of urine pharmacokinetics
Ae, CLr, %Fx
Time frame: 0-4, 4-10, 10-12, 12-24h post dose
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