For Healthcare Professionals

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

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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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Key Inclusion Criteria:


  1. Signed and dated informed consent
  2. Male or female participants aged 18 to 75 years at screening
  3. Have an established diagnosis of PXE
  4. Fully vaccinated for coronavirus disease 2019 (COVID-19) per current Center for Disease Control and Prevention guidelines

EXCLUSION CRITERIA

Key Exclusion Criteria:


  1. Have a history of bone fracture in the past 6 months
  2. Have a history of active metabolic bone disease, excluding osteopenia or osteoporosis without fragility fracture
  3. Have a history of calcium pyrophosphate deposit disease
  4. Have a history of hypophosphatasia
  5. Have a history of untreated hyperparathyroidism
  6. Participated in another interventional research study in the past 60 days.
  7. Used bisphosphonate in the preceding 12 months or had plans to use bisphosphonate during the study.
  8. Received Vitamin B6 supplementation >5 mg/day in the month prior to screening and during the study
  9. Initiated or changed dose of Vitamin D in the preceding month prior to screening
  10. Have an alkaline phosphatase
  11. Have a QTcF interval duration >450 ms at screening
  12. Have moderate to severe renal insufficiency
  13. Are pregnant or breast-feeding women
  14. Are female participants unwilling to use contraceptive methods
  15. Have any elective surgery planned during the study period
  16. 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

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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

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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