A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION 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
EXCLUSION CRITERIA
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
Study Locations
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How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Pseudoxanthoma Elasticum
Age (in years)
18 - 75
Phase
Phase 2
Participants needed
65
Est. Completion Date
Apr 30, 2024
Treatment type
Interventional
Sponsor
Daiichi Sankyo, Inc.
ClinicalTrials.gov identifier
NCT05569252
Study number
DS1211-A-U201
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