Burosumab in Children and Adolescents With X-linked Hypop... | NCT04419363

Studies
Burosumab in Children and Adolescents With X-linked Hypophosphatemia

NCT04419363

Sponsor

Bicetre Hospital

Status

Unknown status

Last Update Posted

Jun 5, 2020Actual

Enrollment

57Estimated

Phase

Phase 4

Conditions

Rare Diseases
X-linked Hypophosphatemia

Interventions

Burosumab Injection

Countries

France

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

Jun 2020

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 18, 2018Actual

Primary Completion Date

Mar 20, 2019Actual

Completion Date

Sep 16, 2022Estimated

First Submitted Date

May 29, 2020

First Submission Date that Met QC Criteria

Jun 3, 2020

First Posted Date

Jun 5, 2020Actual

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

Jun 3, 2020

Last Update Posted Date

Jun 5, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for <13- and >13-years old children affected with X-linked hypophosphatemia.

57 children with XLH were switched from conventional treatment to burosumab. After 12 months we assessed the efficacy and safety of treatment with burosumab on the whole cohort and separately on the cohort of >13-years old adolescents.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

57Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
The whole cohort	Experimental	Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab	Drug: Burosumab Injection

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Burosumab Injection	Drug	Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab	The whole cohort

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Radiological changes in rachitic lesions evaluated with knee MRI	maximum width of the physis and transverse extent of widening	12 months

Secondary Outcomes

Measure	Description	Time Frame
serum phosphate	mmol/l	12 months
renal phosphate reabsorption	mmol/l	12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

insufficient response or refractory to conventional therapy;
complications of conventional therapy: hypercalciuria and/or nephrocalcinosis, and/or persistent secondary hyperparathyroidism;
need for rapid restoration of phosphate metabolism, e.g., late diagnosis (aged >8 years) and/or preparation for planned orthopaedic surgery.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Year

Maximum Age

20 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Bicetre	Recruiting	Le Kremlin-Bicêtre		94270	France

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
D035583	Rare Diseases
D053098	Familial Hypophosphatemic Rickets
D006961	Hyperparathyroidism

Ancestor Terms

ID	Term
D020969	Disease Attributes
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms
D063730	Rickets, Hypophosphatemic

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000601956	burosumab

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided