A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
NCT ID: NCT05569252
Last Updated: 2024-12-31
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
65 participants
INTERVENTIONAL
2022-10-20
2023-11-21
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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DS-1211b low dose
Participants who will be randomized to receive a DS-1211 tablet once daily for 12 weeks.
DS-1211b
DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal
DS-1211b middle dose
Participants who will be randomized to receive a DS-1211b tablet once daily for 12 weeks.
DS-1211b
DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal
DS-1211b high dose
Participants who will be randomized to receive a DS-1211b tablet once daily for 12 weeks.
DS-1211b
DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal
Placebo
Participants who will be randomized to receive a placebo tablet once daily for 12 weeks.
Placebo
Placebo tablet administered once daily in the morning either in the fasted state or with a meal
Interventions
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DS-1211b
DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal
Placebo
Placebo tablet administered once daily in the morning either in the fasted state or with a meal
Eligibility Criteria
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Inclusion Criteria
* 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
* 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 \<lower limit of normal (LLN) range
* 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
18 Years
75 Years
ALL
No
Sponsors
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PXE International
OTHER
Daiichi Sankyo
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Director
Role: STUDY_DIRECTOR
Daiichi Sankyo
Locations
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Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, United States
Boston Neuro Research Center
North Dartmouth, Massachusetts, United States
Infinity Medical Research Inc
North Dartmouth, Massachusetts, United States
Frontage Clinical Services, Inc.
Secaucus, New Jersey, United States
Clinilabs
New York, New York, United States
Clinical Research of Philadelphia, LLC
Philadelphia, Pennsylvania, United States
UMC Utrecht
Utrecht, , Netherlands
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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2022-000676-19
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
DS1211-A-U201
Identifier Type: -
Identifier Source: org_study_id