Patient Reported Outcomes in Adults With Pediatric-onset... | NCT04195763

Studies
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)

NCT04195763

Sponsor

Alexion Pharmaceuticals, Inc.

Status

Completed

Last Update Posted

Jul 10, 2024Actual

Enrollment

50Actual

Phase

Not provided

Conditions

Hypophosphatasia

Interventions

asfotase alfa

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2024

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 6, 2019Actual

Primary Completion Date

Apr 12, 2024Actual

Completion Date

Apr 12, 2024Actual

First Submitted Date

Dec 10, 2019

First Submission Date that Met QC Criteria

Dec 10, 2019

First Posted Date

Dec 12, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 9, 2024

Last Update Posted Date

Jul 10, 2024Actual

Name	Class
Xcenda, LLC	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.

Detailed Description

Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized.

This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

50Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Participants with Pediatric-onset HPP		Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.	Drug: asfotase alfa

Interventions

Name	Type	Description	Arm Group Labels	Other Names
asfotase alfa	Drug	This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores		Baseline, up to 12 months

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

≥ 18 years old
Clinical diagnosis of pediatric-onset HPP
Naïve to asfotase alfa
Expected to begin treatment with asfotase alfa for HPP
Registered in OneSource
Willing and able to provide voluntary, verbal informed consent to participate in this study

Exclusion Criteria:

Pregnant or breastfeeding
Unable to speak and understand English
Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

99 Years

Standard Ages

AdultOlder Adult

Study Population

Male or female participants ≥18 years of age who have been diagnosed with pediatric-onset HPP, have been newly prescribed treatment with asfotase alfa, and are registered in the patient support program managed by OneSource will be invited to participate.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Xcenda, LLC		Palm Harbor	Florida	34685	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D007014	Hypophosphatasia

Ancestor Terms

ID	Term
D008664	Metal Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C570710	asfotase alfa

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided