6 結果
Background: Lymphangioleiomyomatosis (LAM) is characterized by cystic lung destruction, kidney angiomyolipomas (AMLs), and LAM cell growth within the axial lymphatics and multiple other organs and surfaces. LAM occurs both sporadically and in association with tuberous sclerosis complex (TSC).
The objective of this study was to evaluate the accuracy of the CIR-24 compared to caffeine concentration biomarkers in urine and a caffeinated beverage intake frequency screener (CBQ) among a young adult population.
A 24-hour Caffeine Intake Recall (CIR-24) was developed to estimate caffeine
1. Primary objectives:
· To determine the maximum tolerated dose (MTD) of CN128 for single oral administration in thalassemia patients aged 16 and above
· To study the pharmacokinetics of CN128 in thalassemia patients aged 16 and above
2. Design:
The study is designed as a randomized, double-blind,
1. Primary objectives:
- To study the tolerance and safety of multiple oral administration of CN128 in patients with thalassemia aged 16 years and above.
- To study the pharmacokinetics of CN128 in thalassemia patients aged 16 and above by multiple oral administrations of CN128
2. Design:
The study
1. Study Objectives
1.1 Primary objective: The primary objective of this Phase IIa study is to establish clinical Proof-of-Concept (POC) by providing clinically and statistically significant evidence that GIC-1001 is safe and effective in managing visceral pain in male and female patients who