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

• Status: Not yet recruiting
• Trial end date: 2023-09-29
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Daiichi Sankyo, Inc.
• Collaborator: PXE International

Criteria

Key Inclusion Criteria:

• Signed and dated informed consent

• Male or female participants aged 18 to 75 years at screening

• Have an established diagnosis of PXE

• Fully vaccinated for coronavirus disease 2019 (COVID-19) per current Center for Disease Control and Prevention guidelines

Key Exclusion Criteria:

• Have a history of bone fracture in the past 6 months

• Have a history of active metabolic bone disease, excluding osteopenia or osteoporosis without fragility fracture

• Have a history of calcium pyrophosphate deposit disease

• Have a history of hypophosphatasia

• Have a history of untreated hyperparathyroidism

• Participated in another interventional research study in the past 60 days.

• Used bisphosphonate in the preceding 12 months or had plans to use bisphosphonate during the study.

• Received Vitamin B6 supplementation >5 mg/day in the month prior to screening and during the study

• Initiated or changed dose of Vitamin D in the preceding month prior to screening

• Have an alkaline phosphatase- Have a QTcF interval duration >450 ms at screening

• Have moderate to severe renal insufficiency

• Are pregnant or breast-feeding women

• Are female participants unwilling to use contraceptive methods

• Have any elective surgery planned during the study period

• Have any other significant condition (medical, psychiatric, social, or medication) that, in the judgment of the Investigator, would prevent full participation or would be inappropriate for the study