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Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Primary Purpose

Rare Diseases, X-linked Hypophosphatemia

Status

Unknown status

Phase

Phase 4

Locations

France

Study Type

Interventional

Intervention

Burosumab Injection

About this trial

This is an interventional treatment trial for Rare Diseases focused on measuring X-linked hypophosphataemia, fibroblast growth factor 23, burosumab, hyperparathyroidism

Eligibility Criteria

1 Year - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Hospital BicetreRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

The whole cohort

Arm Description

Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab

Outcomes

Primary Outcome Measures

Radiological changes in rachitic lesions evaluated with knee MRI

maximum width of the physis and transverse extent of widening

Secondary Outcome Measures

serum phosphate

mmol/l

renal phosphate reabsorption

mmol/l

alkaline phosphatase

U/l

1,25(OH)vitaminD

pg/ml

parathyroid hormone

ng/l

height

standard deviation score

functional capacity

6-minute walk test, standard deviation score

incidence of dental abcesses

dental examination

incidence of hearing problems

ORL examination, audiogramm

incidence of neurological problems (craniosynostosis, Chiari I malformation)

neurosurgical examination and brain MRI

incidence of nephrocalcinosis

renal ultrasound

incidence of hyperparathyroidism

blood levels of parathyroid hormone

incidence of any side effects

registration of any side effects during the treatment by telephone call

Full Information

NCT ID

NCT04419363

First Posted

May 29, 2020

Last Updated

June 3, 2020

Sponsor

Bicetre Hospital

1. Study Identification

Unique Protocol Identification Number

NCT04419363

Brief Title

Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Official Title

12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study

Study Type

Interventional

2. Study Status

Record Verification Date

June 2020

Overall Recruitment Status

Unknown status

Study Start Date

March 18, 2018 (Actual)

Primary Completion Date

March 20, 2019 (Actual)

Study Completion Date

September 16, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Bicetre Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

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

Rare Diseases, X-linked Hypophosphatemia

Keywords

X-linked hypophosphataemia, fibroblast growth factor 23, burosumab, hyperparathyroidism

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

57 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

The whole cohort

Arm Type

Experimental

Arm Description

Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab

Intervention Type

Drug

Intervention Name(s)

Burosumab Injection

Intervention Description

Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab

Primary Outcome Measure Information:

Title

Radiological changes in rachitic lesions evaluated with knee MRI

Description

maximum width of the physis and transverse extent of widening

Time Frame

12 months

Secondary Outcome Measure Information:

Title

serum phosphate

Description

mmol/l

Time Frame

12 months

Title

renal phosphate reabsorption

Description

mmol/l

Time Frame

12 months

Title

alkaline phosphatase

Description

U/l

Time Frame

12 months

Title

1,25(OH)vitaminD

Description

pg/ml

Time Frame

12 months

Title

parathyroid hormone

Description

ng/l

Time Frame

12 months

Title

height

Description

standard deviation score

Time Frame

12 months

Title

functional capacity

Description

6-minute walk test, standard deviation score

Time Frame

12 months

Title

incidence of dental abcesses

Description

dental examination

Time Frame

12 months

Title

incidence of hearing problems

Description

ORL examination, audiogramm

Time Frame

12 months

Title

incidence of neurological problems (craniosynostosis, Chiari I malformation)

Description

neurosurgical examination and brain MRI

Time Frame

12 months

Title

incidence of nephrocalcinosis

Description

renal ultrasound

Time Frame

12 months

Title

incidence of hyperparathyroidism

Description

blood levels of parathyroid hormone

Time Frame

12 months

Title

incidence of any side effects

Description

registration of any side effects during the treatment by telephone call

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

20 Years

Accepts Healthy Volunteers

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.

Facility Information:

Facility Name

Hospital Bicetre

City

Le Kremlin-Bicêtre

ZIP/Postal Code

94270

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Volha Zhukouskaya, MD, PhD

Phone

+33766681018

vzhukuskaya@genethon.fr

First Name & Middle Initial & Last Name & Degree

Agnès Linglart, MD, PhD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Burosumab in Children and Adolescents With X-linked Hypophosphatemia

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