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Rare DiseasesPhase 4NCT04419363

Burosumab in Children and Adolescents With X-linked Hypophosphatemia

A Phase 4 study testing Burosumab Injection in people with Rare Diseases and X-linked Hypophosphatemia, run by Bicetre Hospital at 1 site in France.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 20 years
Conditions the study is for
Rare Diseases, X-linked Hypophosphatemia
What is being tested?
Burosumab Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
57 participants
Where
1 siteFrance
Study dates
18 Mar 2018to 16 Sep 2022
Phase
Phase 4
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

France1 site, 1 recruiting

  • Hospital Bicetre

    Le Kremlin-Bicêtre, 94270

    Volha Zhukouskaya, MD, PhD, +33766681018, vzhukuskaya@genethon.fr

    Agnès Linglart, MD, PhD

    Recruiting

Other trials in France

About this study

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

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.

Being tested
Burosumab Injection
Keywords
X-linked hypophosphataemia, fibroblast growth factor 23, burosumab, hyperparathyroidism
Medical subject headings
Rare Diseases, Familial Hypophosphatemic Rickets, Hyperparathyroidism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Burosumab InjectionDrug

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

Study groups in NCT04419363
Study groupRoleWhat they receive
The whole cohortExperimentalChildren affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab
Receives: Drug: Burosumab Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Radiological changes in rachitic lesions evaluated with knee MRI

    maximum width of the physis and transverse extent of widening

    Measured: 12 months

Secondary outcomes (13)

  1. serum phosphate

    mmol/l

    Measured: 12 months

  2. renal phosphate reabsorption

    mmol/l

    Measured: 12 months

  3. alkaline phosphatase

    U/l

    Measured: 12 months

  4. 1,25(OH)vitaminD

    pg/ml

    Measured: 12 months

  5. parathyroid hormone

    ng/l

    Measured: 12 months

  6. height

    standard deviation score

    Measured: 12 months

  7. functional capacity

    6-minute walk test, standard deviation score

    Measured: 12 months

  8. incidence of dental abcesses

    dental examination

    Measured: 12 months

  9. incidence of hearing problems

    ORL examination, audiogramm

    Measured: 12 months

  10. incidence of neurological problems (craniosynostosis, Chiari I malformation)

    neurosurgical examination and brain MRI

    Measured: 12 months

  11. incidence of nephrocalcinosis

    renal ultrasound

    Measured: 12 months

  12. incidence of hyperparathyroidism

    blood levels of parathyroid hormone

    Measured: 12 months

  13. incidence of any side effects

    registration of any side effects during the treatment by telephone call

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Jun 2020
  2. Study start18 Mar 2018
  3. Primary completion20 Mar 2019
  4. Study completion16 Sep 2022 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
57 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04419363
Sponsor's protocol ID
20200528104746
Status last verified
Jun 2020
First submitted
29 May 2020
Last updated
5 Jun 2020
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Burosumab Injection in people with Rare Diseases and X-linked Hypophosphatemia, run by Bicetre Hospital at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 5 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Radiological changes in rachitic lesions evaluated with knee MRI (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04419363.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2020.

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