A phase 2, 12-week, randomized, double-blind, placebo-controlled study of DS-1211b in individuals with pseudoxanthoma elasticum

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 16

Inclusion Criteria

1. Sign and date the ICF prior to the start of any study-specific qualification
procedures.
2. Are male or female participants aged 18 to 75 years at screening.
3. Have an established diagnosis of PXE, with typical ocular and dermatological
clinical features:
a. Ocular findings - angioid streaks or peau d*orange
b. Skin findings - characteristic PXE papules and plaques or diagnostic
histopathological changes in lesional skin
4. Are fully vaccinated for coronavirus disease 2019 (COVID-19) per current
Center for Disease Control and Prevention guidelines
5. For women of childbearing potential (not postmenopausal as a result of
either natural or postsurgery cessation of menses), must have a negative serum
pregnancy test at screening and must be willing to use an effective method of
birth control, as detailed in Section 17.1.2 of the protocol upon entering
study screening, during the treatment period, and up until the time of the
follow-up visit.
6. Are willing and able to comply with scheduled visits, drug administration
plan,
laboratory tests, other study procedures, and study restrictions.

Exclusion Criteria

1. Have a history of bone fracture in the past 6 months.
2. Have a history of active metabolic bone disease that significantly affect
the interpretation of study biomarker results, excluding osteopenia or
osteoporosis without fragility fracture.
3. Have a history of calcium pyrophosphate deposit disease such as
chondrocalcinosis, pseudogout, and pyrophosphate arthropathy.
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. Are participating or have participated within the last 12 months in PXE
trials (eg, trials with PPi, PPi analogues such as bisphosphonate, or ENPP1 and
its analogues) or in clinical trials relating to bone mineralization.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Safety parameters: Incidence of adverse events(AEs), including serious adverse<br /><br>events (SAEs), and treatment-emergent AEs, physical<br /><br>examination/assessment findings including vital sign measurements, standard<br /><br>clinical laboratory parameters, and electrocardiograms.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>PD endpoints: AProfiles of ALP, inorganic pyrophosphate (PPi), and pyridoxal 5*-<br /><br>phosphate (PLP)<br /><br><br /><br>PK endpoints: Plasma concentration and PK parameters of DS-1211a Include, but<br /><br>are not limited to, maximum plasma concentration, time to reach maximum plasma<br /><br>concentration, trough plasma concentration, area under the plasma<br /><br>concentration-time curve during dosing interval, area under the plasma<br /><br>concentration-time curve up to the last uantifiable time, and area under the<br /><br>plasma concentration-time curve up to infinity (if possible to calculate).</p><br>