Phase 2
N=65
A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
Pseudoxanthoma Elasticum
Bottom Line
View on ClinicalTrials.gov: NCT05569252 ↗Enrolled (actual)
65
Serious AEs
1.5%
Results posted
Dec 2024
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b — 8; 7; 6; 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- DS-1211b (Drug); Placebo (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Daiichi Sankyo
- Primary completion
- Nov 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b |
8; 7; 6; 6; 0; 0 | — |
| PRIMARY Percent Change From Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels |
17.93; 24.39; 39.40; -0.48; 25.02; 42.89 | — |
| PRIMARY Percent Change From Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels |
118.97; 11.32; 23.95; 13.33; 17.75; -5.55 | — |
| PRIMARY Percent Change From Baseline in Pharmacodynamic Parameter Pyridoxal 5'-Phosphate (PLP) Levels |
11.01; 45.42; 50.97; 3.42; 8.56; 36.73 | — |
| SECONDARY Pharmacokinetic Parameter Maximum Concentration (Cmax) |
252; 540; 713; 255; 547; 726 | — |
| SECONDARY Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) |
1.25; 1.13; 1.25; 1.25; 1.13; 1.25 | — |
| SECONDARY Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough) |
17.15; 26.30; 48.43; 15.19; 38.53; 54.48 | — |
| SECONDARY Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC) |
978; 2090; 2780; 1020; 2210; 3000 | — |
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.
Eligibility 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 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
Data sourced from ClinicalTrials.gov (NCT05569252). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.