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Burosumab in Children and Adolescents With X-linked Hypophosphatemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04419363
Recruitment Status : Recruiting
First Posted : June 5, 2020
Last Update Posted : June 5, 2020
Information provided by (Responsible Party):
Volha Zhukouskaya, Bicetre Hospital

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.

Condition or disease Intervention/treatment Phase
Rare Diseases X-linked Hypophosphatemia Drug: Burosumab Injection Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 57 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study
Actual Study Start Date : March 18, 2018
Actual Primary Completion Date : March 20, 2019
Estimated Study Completion Date : September 16, 2022

Arm Intervention/treatment
Experimental: The whole cohort
Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab
Drug: Burosumab Injection
Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab

Primary Outcome Measures :
  1. Radiological changes in rachitic lesions evaluated with knee MRI [ Time Frame: 12 months ]
    maximum width of the physis and transverse extent of widening

Secondary Outcome Measures :
  1. serum phosphate [ Time Frame: 12 months ]

  2. renal phosphate reabsorption [ Time Frame: 12 months ]

  3. alkaline phosphatase [ Time Frame: 12 months ]

  4. 1,25(OH)vitaminD [ Time Frame: 12 months ]

  5. parathyroid hormone [ Time Frame: 12 months ]

  6. height [ Time Frame: 12 months ]
    standard deviation score

  7. functional capacity [ Time Frame: 12 months ]
    6-minute walk test, standard deviation score

  8. incidence of dental abcesses [ Time Frame: 12 months ]
    dental examination

  9. incidence of hearing problems [ Time Frame: 12 months ]
    ORL examination, audiogramm

  10. incidence of neurological problems (craniosynostosis, Chiari I malformation) [ Time Frame: 12 months ]
    neurosurgical examination and brain MRI

  11. incidence of nephrocalcinosis [ Time Frame: 12 months ]
    renal ultrasound

  12. incidence of hyperparathyroidism [ Time Frame: 12 months ]
    blood levels of parathyroid hormone

  13. incidence of any side effects [ Time Frame: 12 months ]
    registration of any side effects during the treatment by telephone call

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Ages Eligible for Study:   1 Year to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04419363

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Hospital Bicetre Recruiting
Le Kremlin-Bicêtre, France, 94270
Contact: Volha Zhukouskaya, MD, PhD    +33766681018   
Principal Investigator: Agnès Linglart, MD, PhD         
Sponsors and Collaborators
Bicetre Hospital
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Volha Zhukouskaya, MD, PhD, Bicetre Hospital Identifier: NCT04419363    
Other Study ID Numbers: 20200528104746
First Posted: June 5, 2020    Key Record Dates
Last Update Posted: June 5, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Volha Zhukouskaya, Bicetre Hospital:
X-linked hypophosphataemia
fibroblast growth factor 23
Additional relevant MeSH terms:
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Familial Hypophosphatemic Rickets
Rare Diseases
Disease Attributes
Pathologic Processes
Phosphorus Metabolism Disorders
Metabolic Diseases
Rickets, Hypophosphatemic
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Calcium Metabolism Disorders
Vitamin D Deficiency
Deficiency Diseases
Nutrition Disorders