NCT04419363

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
57

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Mar 2018

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 18, 2018

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 20, 2019

Completed
1.2 years until next milestone

First Submitted

Initial submission to the registry

May 29, 2020

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 5, 2020

Completed
2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 16, 2022

Completed
Last Updated

June 5, 2020

Status Verified

June 1, 2020

Enrollment Period

1 year

First QC Date

May 29, 2020

Last Update Submit

June 3, 2020

Conditions

Rare DiseasesX-linked Hypophosphatemia

Keywords

X-linked hypophosphataemiafibroblast growth factor 23burosumabhyperparathyroidism

Outcome Measures

Primary Outcomes (1)

  • Radiological changes in rachitic lesions evaluated with knee MRI

    maximum width of the physis and transverse extent of widening

    12 months

Secondary Outcomes (13)

  • serum phosphate

    12 months

  • renal phosphate reabsorption

    12 months

  • alkaline phosphatase

    12 months

  • 1,25(OH)vitaminD

    12 months

  • parathyroid hormone

    12 months

  • +8 more secondary outcomes

Study Arms (1)

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

Burosumab InjectionDRUG

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

The whole cohort

Eligibility Criteria

Age1 Year - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital Bicetre

Le Kremlin-Bicêtre, 94270, France

RECRUITING

MeSH Terms

Conditions

Rare DiseasesFamilial Hypophosphatemic RicketsHyperparathyroidism

Interventions

burosumab

Condition Hierarchy (Ancestors)

Disease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsRickets, HypophosphatemicRicketsBone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesHypophosphatemia, FamilialRenal Tubular Transport, Inborn ErrorsKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesMetal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesCalcium Metabolism DisordersHypophosphatemiaPhosphorus Metabolism DisordersVitamin D DeficiencyAvitaminosisDeficiency DiseasesMalnutritionNutrition DisordersParathyroid DiseasesEndocrine System Diseases

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

May 29, 2020

First Posted

June 5, 2020

Study Start

March 18, 2018

Primary Completion

March 20, 2019

Study Completion

September 16, 2022

Last Updated

June 5, 2020

Record last verified: 2020-06

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)