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Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)

NCT02163577 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2024-05-06

Study results available
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Summary

The objectives of the study are to:

* Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants
* Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile
* Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients
* Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients
* Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function
* Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients
* Evaluate the long-term safety and efficacy of burosumab

Conditions

  • X-linked Hypophosphatemia

Interventions

BIOLOGICAL

burosumab

solution for SC injection

Sponsors & Collaborators

  • Kyowa Kirin Co., Ltd.

    collaborator INDUSTRY

  • Kyowa Kirin, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
5 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-07-02
Primary Completion
2018-10-30
Completion
2018-10-30

Countries

  • United States
  • France
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02163577 on ClinicalTrials.gov