NCT05569252

A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum

A Phase 2, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study of DS-1211b in Individuals With PseudoXanthoma Elasticum

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Daiichi Sankyo · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This study was designed to evaluate the safety, tolerability, pharmacodynamics (PD) of DS-1211b, and pharmacokinetics (PK) in individuals with Pseudoxanthoma elasticum (PXE). PXE is a rare disease that is associated with significant risks of visual impairments and comorbidity from peripheral and cardiovascular diseases, and adversely impacts the quality of life in afflicted individuals.

Detailed description

DS-1211b, a potent small-molecule inhibitor of tissue-nonspecific alkaline phosphatase, is being developed for the treatment ectopic calcification diseases such as PXE. This study will assess DS-1211b (low-, middle-, and high-dose tablets) administered once daily for 12 weeks in individuals with PXE.

Conditions

• Pseudoxanthoma Elasticum

Interventions

Type	Name	Description
DRUG	DS-1211b	DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal
OTHER	Placebo	Placebo tablet administered once daily in the morning either in the fasted state or with a meal

Timeline

• Start date: 2022-10-20
• Primary completion: 2023-11-21
• Completion: 2023-11-21
• First posted: 2022-10-06
• Last updated: 2024-12-31
• Results posted: 2024-12-31

Locations

7 sites across 2 countries: United States, Netherlands

Regulatory

• FDA-regulated drug study