Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia (IBIS XLH)

May 19, 2026 updated by: Novo Nordisk A/S

Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia - A Prospective Observational Cohort Study - IBIS XLH

The purpose of this study is to better understand how X-linked hypophosphatemia (XLH) affects the body and daily life. Phosphate levels are critical in managing XLH, we aim to study how these levels change in patients taking burosumab and how they relate to bone health, overall disease burden and XLH-related conditions.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • St Leonards, New South Wales, Australia, 2065
        • Northern Sydney Cancer Centre-Royal North Shore Hospital (RNSH)
      • Westmead, New South Wales, Australia, 2145
        • Westmead Hospital
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Monash University - Monash Medical Centre (MMC)
  • Canada
    • Ontario
      • Oakville, Ontario, Canada, L6M 1M1
        • Bone Research and Education Centre
  • France
      • Le Kremlin, France, 94275
        • CHU de Bicetre
      • Paris, France, 75014
        • Hopital Cochin - APHP
  • Germany
      • Berlin, Germany, 13353
        • Charite Campus Virchow
      • Göttingen, Germany, 37075
        • Endokrinologikum Goettingen
      • Wuezburg, Germany, 97074
        • Universitat Wurzburg Koenig-Ludwig-Haus
  • Italy
      • Milan, Italy, 20145
        • Istituto Auxologico Italiano
      • Padova, Italy, 35128
        • Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia
      • Rome, Italy, 00128
        • Fondazione Policlinico Universitario Campus Bio-medico
    • Florence
      • Sesto Fiorentino, Florence, Italy, 50019
        • Office of Francesco Di Costanzo, MD
  • Japan
      • Osaka, Japan, 553-0003
        • Japan Community Healthcare Organization Osaka Hospital
      • Osaka, Japan, 530-0052
        • Iseikai International General Hospital
    • Fukuoka
      • Fukuoka, Fukuoka, Japan, 814-0180
        • Fukuoka University - Fukuoka University Hospital
    • Okayama-ken
      • Okayama, Okayama-ken, Japan, 700-0013
        • Okayama Saiseikai Outpatient Center Hospital
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-8655
        • The University of Tokyo Hospital
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leiden University Medical Center (Leids Universitair Medisch Centrum (LUMC))
  • United States
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5271
        • Indiana University Hospital
    • Ohio
      • Columbus, Ohio, United States, 43203
        • The Ohio State University Wexner Medical Center (OSUWMC) - CarePoint East
    • Tennessee
      • Nashville, Tennessee, United States, 37232-8148
        • Vanderbilt University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Adults with XLH, confirmed genetically or clinically as determined by the investigator, who have been treated with burosumab for at least 90 days prior to the screening/baseline visit and whose treatment is still ongoing. Participants will be followed for a maximum of three years, continuing until end of trial visit, withdrawal of consent or loss to follow-up, whichever occurs first.

Description

Inclusion Criteria:

  • Informed consent obtained before any trial-related activities, i.e., any procedure related to recording of data according to the protocol.
  • Male or female and aged 18-64 years (both inclusive) at the time of signing informed consent.
  • Diagnosis of congenital XLH, confirmed genetically or clinically as determined by the investigator, supported by documentation.
  • Participants have been treated with burosumab for at least 90 days prior to the screening/baseline visit and treatment is still ongoing.
  • Participant confirms ability and willingness to attend visits according to routine clinical care and mandatory assessments at the site.

Exclusion Criteria:

  • Previous rescreening for this trial.
  • Current participation in any interventional clinical trial. Participation in another non-interventional trial, such as a patient registry trial, is permitted.
  • Any condition not associated with XLH, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Adults with XLH
Adults with XLH, confirmed genetically or clinically as determined by the investigator, who have been treated with burosumab for at least 90 days prior to the screening/baseline visit and whose treatment is still ongoing will be followed for a maximum of three years, continuing until end of trial visit, withdrawal of consent or loss to follow-up, whichever occurs first.
Other: No treatment given
No treatment given

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Average Trough Serum Phosphate Concentration
Time Frame: From baseline to last assessment (Up to 3 years)
Average trough serum phosphate concentration, defined as the area under the concentration-time curve (AUC) divided by the total duration of the assessment period (AUC divided by time). Measured in milligrams per deciliter (mg/dL).
From baseline to last assessment (Up to 3 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Investigators

  • Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

July 7, 2026

Primary Completion (Estimated)

July 7, 2030

Study Completion (Estimated)

July 7, 2030

Study Registration Dates

First Submitted

May 19, 2026

First Submitted That Met QC Criteria

May 19, 2026

First Posted (Actual)

May 26, 2026

Study Record Updates

Last Update Posted (Actual)

May 26, 2026

Last Update Submitted That Met QC Criteria

May 19, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NN8661-8325

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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