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

Primary Purpose

Pseudoxanthoma Elasticum

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

DS-1211b

Placebo

About this trial

This is an interventional treatment trial for Pseudoxanthoma Elasticum focused on measuring Pseudoxanthoma Elasticum, DS-1211b

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Mid-Atlantic Epilepsy and Sleep Center
Boston Neuro Research Center
Infinity Medical Research Inc
Frontage Clinical Services, Inc.
Clinilabs
Clinical Research of Philadelphia, LLC
UMC Utrecht

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Arm Label

DS-1211b low dose

DS-1211b middle dose

DS-1211b high dose

Placebo

Arm Description

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

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

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

Outcomes

Primary Outcome Measures

Number of Participants with Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b

Change from Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels

Change from Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels

Change from Baseline in Pharmacodynamic Parameter Pyridoxal 5'-phosphate (PLP) Levels

Secondary Outcome Measures

Pharmacokinetic Parameter Maximum Concentration (Cmax)

Pharmacokinetic Parameter Time to Maximum Concentration (Tmax)

Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough)

Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC)

Full Information

NCT ID

NCT05569252

First Posted

October 4, 2022

Last Updated

October 2, 2023

Sponsor

Daiichi Sankyo, Inc.

Collaborators

PXE International

1. Study Identification

Unique Protocol Identification Number

NCT05569252

Brief Title

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

Official Title

A Phase 2, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study of DS-1211b in Individuals With PseudoXanthoma Elasticum

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

October 20, 2022 (Actual)

Primary Completion Date

April 2024 (Anticipated)

Study Completion Date

April 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Daiichi Sankyo, Inc.

Collaborators

PXE International

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief 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.

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pseudoxanthoma Elasticum

Keywords

Pseudoxanthoma Elasticum, DS-1211b

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

65 (Actual)

8. Arms, Groups, and Interventions

Arm Title

DS-1211b low dose

Arm Type

Experimental

Arm Description

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

Arm Title

DS-1211b middle dose

Arm Type

Experimental

Arm Description

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

Arm Title

DS-1211b high dose

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

DS-1211b

Intervention Description

DS-1211b tablet administered once daily in the morning either in the fasted state or with a meal

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Placebo tablet administered once daily in the morning either in the fasted state or with a meal

Primary Outcome Measure Information:

Title

Number of Participants with Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b

Time Frame

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

Title

Change from Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels

Time Frame

Screening (Days -1 to -30) and at Days 1, 15, 43, 84, 86-88, and 98 post-dose of 12-week treatment period

Title

Change from Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels

Time Frame

Screening (Days -1 to -30) and at Days 1, 15, 43, 84

Title

Change from Baseline in Pharmacodynamic Parameter Pyridoxal 5'-phosphate (PLP) Levels

Time Frame

Screening (Days -1 to -30) and at Days 1, 15, 43, 84, 86-88, and 98 post-dose of 12-week treatment period

Secondary Outcome Measure Information:

Title

Pharmacokinetic Parameter Maximum Concentration (Cmax)

Time Frame

Days, 1, 15, 43, and 84

Title

Pharmacokinetic Parameter Time to Maximum Concentration (Tmax)

Time Frame

Days, 1, 15, 43, and 84

Title

Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough)

Time Frame

Days, 1, 15, 43, and 84

Title

Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC)

Time Frame

Days, 1, 15, 43, and 84

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

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 <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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Director

Organizational Affiliation

Daiichi Sankyo, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Mid-Atlantic Epilepsy and Sleep Center

City

Bethesda

State/Province

Maryland

ZIP/Postal Code

20817

Country

United States

Facility Name

Boston Neuro Research Center

City

North Dartmouth

State/Province

Massachusetts

ZIP/Postal Code

02747

Country

United States

Facility Name

Infinity Medical Research Inc

City

North Dartmouth

State/Province

Massachusetts

ZIP/Postal Code

02747

Country

United States

Facility Name

Frontage Clinical Services, Inc.

City

Secaucus

State/Province

New Jersey

ZIP/Postal Code

07094

Country

United States

Facility Name

Clinilabs

City

New York

State/Province

New York

ZIP/Postal Code

10019

Country

United States

Facility Name

Clinical Research of Philadelphia, LLC

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19114

Country

United States

Facility Name

UMC Utrecht

City

Utrecht

ZIP/Postal Code

3584

Country

Netherlands

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

De-identified individual participant data (IPD) on completed studies and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

IPD Sharing Time Frame

Completed studies that has reached a global end or completion with all data set collected and analyzed, and for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.

IPD Sharing Access Criteria

Formal request from qualified scientific and medical researchers on IPD and clinical study documents on completed clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.

IPD Sharing URL

https://vivli.org/ourmember/daiichi-sankyo/

Learn more about this trial

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

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