Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia

NCT07607600 | Not Yet Recruiting

• 1 other identifier: NN8661-8325
• Study Type: observational
• Target: 100
• Locations: 8 countries
• Sites: 23

Brief Summary

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.

Conditions

X-linked Hypophosphatemia (XLH)

Outcome Measures

Primary Outcomes (1)

• Average Trough Serum Phosphate Concentration

  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)

Study Arms (1)

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

Interventions

No treatment given OTHER

No treatment given

Adults with XLH

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (23)

Yale University School of Medicine

New Haven, Connecticut, 06510, United States

Location

Indiana University Hospital

Indianapolis, Indiana, 46202-5271, United States

Location

The Ohio State University Wexner Medical Center (OSUWMC) - CarePoint East

Columbus, Ohio, 43203, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-8148, United States

Location

Northern Sydney Cancer Centre-Royal North Shore Hospital (RNSH)

St Leonards, New South Wales, 2065, Australia

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Monash University - Monash Medical Centre (MMC)

Clayton, Victoria, 3168, Australia

Location

Bone Research and Education Centre

Oakville, Ontario, L6M 1M1, Canada

Location

CHU de Bicetre

Le Kremlin, 94275, France

Location

Hopital Cochin - APHP

Paris, 75014, France

Location

Charite Campus Virchow

Berlin, 13353, Germany

Location

Endokrinologikum Goettingen

Göttingen, 37075, Germany

Location

Universitat Wurzburg Koenig-Ludwig-Haus

Wuezburg, 97074, Germany

Location

Office of Francesco Di Costanzo, MD

Sesto Fiorentino, Florence, 50019, Italy

Location

Istituto Auxologico Italiano

Milan, 20145, Italy

Location

Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia

Padova, 35128, Italy

Location

Fondazione Policlinico Universitario Campus Bio-Medico

Rome, 00128, Italy

Location

Fukuoka University - Fukuoka University Hospital

Fukuoka, Fukuoka, 814-0180, Japan

Location

Okayama Saiseikai Outpatient Center Hospital

Okayama, Okayama-ken, 700-0013, Japan

Location

The University of Tokyo Hospital

Bunkyo-ku, Tokyo, 113-8655, Japan

Location

Iseikai International General Hospital

Osaka, 530-0052, Japan

Location

Japan Community Healthcare Organization Osaka Hospital

Osaka, 553-0003, Japan

Location

Leiden University Medical Center (Leids Universitair Medisch Centrum (LUMC))

Leiden, 2333 ZA, Netherlands

Location

MeSH Terms

Conditions

Familial Hypophosphatemic Rickets

Condition Hierarchy (Ancestors)

Rickets, Hypophosphatemic; Rickets; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Hypophosphatemia, Familial; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Hypophosphatemia; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Study Officials

• Clinical Transparency dept. 2834

  Novo Nordisk A/S

  STUDY DIRECTOR

Central Study Contacts

Novo Nordisk

CONTACT

(+1) 866-867-7178; clinicaltrials@novonordisk.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2026
• First Posted: May 26, 2026
• Study Start: July 7, 2026
• Primary Completion (Estimated): July 7, 2030
• Study Completion (Estimated): July 7, 2030
• Last Updated: May 26, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (4); CA (1); DE (3); IT (4); NL (1); FR (2); JP (5); AU (3)