CompletedPhase 2

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

United States, France52 participantsStarted 2014-07-02

Plain-language 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

Who can participate

Age range5 Years – 12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female, aged 5 - 12 years, inclusive, with open growth plates
✓. Tanner stage of 2 or less based on breast and testicular development
✓. Diagnosis of XLH supported by ONE of the following:
✓. Biochemical findings associated with XLH including:
✓. Standing height \< 50th percentile for age and gender using local normative data.
✓. Radiographic evidence of active bone disease including rickets in the wrists and/or knees, AND/OR femoral/tibial bowing, OR, for expansion subjects, a Rickets Severity Score (RSS) score in the knee of at least 1.5 as determined by central read.
✓. Willing to provide access to prior medical records for the collection of historical growth, biochemical and radiographic data, and disease history.
✓. Provide written or verbal assent (if possible) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.

Exclusion criteria

What they're measuring

Change From Baseline in RSS Total Score Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in Serum Phosphorus Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in Serum 1,25(OH)2D Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in TmP/GFR Over Time

Timeframe: Baseline, Week 40, 64, 160

Trial details

NCT IDNCT02163577

SponsorKyowa Kirin, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-10-30

Results submitted2019-04-29

View on ClinicalTrials.gov More X-linked Hypophosphatemia trials

Plain-language summary

Who can participate

Age range5 Years – 12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female, aged 5 - 12 years, inclusive, with open growth plates
✓. Tanner stage of 2 or less based on breast and testicular development
✓. Diagnosis of XLH supported by ONE of the following:
✓. Biochemical findings associated with XLH including:
✓. Standing height \< 50th percentile for age and gender using local normative data.
✓. Radiographic evidence of active bone disease including rickets in the wrists and/or knees, AND/OR femoral/tibial bowing, OR, for expansion subjects, a Rickets Severity Score (RSS) score in the knee of at least 1.5 as determined by central read.
✓. Willing to provide access to prior medical records for the collection of historical growth, biochemical and radiographic data, and disease history.
✓. Provide written or verbal assent (if possible) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.

Exclusion criteria

What they're measuring

Change From Baseline in RSS Total Score Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in Serum Phosphorus Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in Serum 1,25(OH)2D Over Time

Timeframe: Baseline, Week 40, 64, 160

Change From Baseline in TmP/GFR Over Time

Timeframe: Baseline, Week 40, 64, 160