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

Daiichi Sankyo

Status and phase: Completed; Phase 2

Conditions: Pseudoxanthoma Elasticum

Treatments: Drug: DS-1211b; Other: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05569252

2022-000676-19 (EudraCT Number)

DS1211-A-U201

Details and patient eligibility

About

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.

Full description

DS-1211b, a potent small-molecule inhibitor of tissue-nonspecific alkaline phosphatase, is being developed for the treatment ectopic calcification diseases such as PXE. This study will assess DS-1211b (low-, middle-, and high-dose tablets) administered once daily for 12 weeks in individuals with PXE.

Enrollment: 65 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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 <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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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65 participants in 4 patient groups, including a placebo group

DS-1211b low dose

Experimental group

Description:

Participants who will be randomized to receive a DS-1211 tablet once daily for 12 weeks.

Treatment:

Drug: DS-1211b

DS-1211b middle dose

Experimental group

Description:

Participants who will be randomized to receive a DS-1211b tablet once daily for 12 weeks.

Treatment:

Drug: DS-1211b

DS-1211b high dose

Experimental group

Description:

Participants who will be randomized to receive a DS-1211b tablet once daily for 12 weeks.

Treatment:

Drug: DS-1211b

Placebo

Placebo Comparator group

Description:

Participants who will be randomized to receive a placebo tablet once daily for 12 weeks.

Treatment:

Other: Placebo