Dosing regimen of therapeutic agent for ataxia associated with spinocerebellar degeneration
A technology for spinocerebellar and ataxia, which can be applied in the direction of pharmaceutical formulations, drug combinations, organic active ingredients, etc., and can solve problems such as increased hormone effects
- Summary
- Abstract
- Description
- Claims
- Application Information
AI Technical Summary
Problems solved by technology
Method used
Image
Examples
Embodiment 1
[0064] Repeated-dose study in healthy adult males
[0065] 1. Test method
[0066] Taking 50 healthy adult males (8 cases in each group and 10 cases in the placebo group) as objects, 0.25, 0.5 or 1.0 mg of lobatirrellin trihydrate or placebo was given once a day, or lobatirrellin Oral administration of 0.25 or 0.5 mg trihydrate or placebo was repeated twice a day for 9 days after breakfast or dinner, respectively.
[0067] 2. Evaluation items
[0068] to FT 3 、FT 4 , TSH and prolactin (PRL) serum concentrations (mean values) and adverse events were evaluated.
[0069] 3. Results
[0070] figure 1 and figure 2 Respectively represent each administration group on the 1st day, the 5th day and the 9th day, FT 3 and FT 4 Changes in concentrations (mean values) in serum.
[0071] About FT 3 Value, the change of 0.25mg twice a day administration group (0.25mg / bid) value compared with the change of 0.5mg once a day administration group (0.5mg / qd) value, the FT of 0.5mg / qd g...
Embodiment 2
[0075] Human pharmacokinetic studies
[0076] Taking 48 healthy adult males (6 cases in each group and 12 cases in the placebo group) as objects, 0.1, 0.3, 1, 2.5, 5 or 10 mg of lobatirrellin trihydrate or placebo were administered once on an empty stomach. medication. Analyzing the pharmacokinetics of the lobatirelin of the prototype drug at this time, the results showed that in the dose range of 0.1 to 10 mg, the Cmax (the highest plasma concentration), AUC and 0-∞ (area under the plasma concentration-time curve from 0 to infinity) and Ae 0-48 (cumulative urine excretion from 0 to 48 hours) was linear.
[0077] From the results of Examples 1 and 2, it can be deduced that the usage of lobatirrellin or its pharmacologically acceptable salt is compared with the usage of dividing the daily dose into twice a day and administering it once a day. The dosing regimen is more likely to reduce the risk of side effects caused by repeated dosing-induced increases in thyroid hormone. ...
Embodiment 3
[0079] Clinical trial for patients with spinocerebellar degeneration (Phase II clinical trial)
[0080] 1. Test method
[0081] Taking 225 patients with spinocerebellar degeneration as subjects, 0.4, 0.8, 1.6 or 3.2 mg of lobatirrellin trihydrate or a placebo was orally administered once a day after breakfast for 24 weeks (double-blind test method).
[0082] 2. Effectiveness evaluation items and safety evaluation items
[0083] The effectiveness evaluation item is set to the SARA total score change (the SARA total score at the final evaluation of the verification period (the final observation value in the verification period) - the SARA total score at the end of the previous observation period), etc., and the safety evaluation item is set to adverse events and the occurrence of side effects, physiological examination (blood pressure, pulse, etc.), endocrinology examination (FT 3 、FT 4 etc.
[0084] In addition, SARA sub-item scores ((gait (0-8 points), standing posture (0-...
PUM
Login to View More Abstract
Description
Claims
Application Information
Login to View More