A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum (NCT05569252) | Clinical Trial Compass
CompletedPhase 2
A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
United States, Netherlands65 participantsStarted 2022-10-20
Plain-language 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.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 \<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 inap…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b
Timeframe: From the date of signing informed consent form up to Day 98 (14 days after last dose of study drug) post-dose of 12-week treatment period
2
Percent Change From Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels
Timeframe: Pre-dose on Days 15, 43, 84; Day 86-88 and Day 98 of 12-week treatment period
3
Percent Change From Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels
Timeframe: Pre-dose on Days 15, 43, and 84 of 12-week treatment period
4
Percent Change From Baseline in Pharmacodynamic Parameter Pyridoxal 5'-Phosphate (PLP) Levels
Timeframe: Pre-dose on Days 15, 43, 84; Day 86-88 and Day 98 of 12-week treatment period