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Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH | Clinical Trials | Clareo Health

COMPLETEDPHASE4

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

An Open-label, Multi-center, Single-cohort, Post-marketing Phase 4 Study to Evaluate the Efficacy, Pharmacodynamics, and Safety of the Anti-FGF23 Antibody, KRN23, in Pediatric Chinese Patients With X-linked Hypophosphatemic Rickets/Osteomalacia (XLH)

NCT04842032•Kyowa Kirin Co., Ltd.

View on ClinicalTrials.gov

Summary

The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH

Eligibility

Age

1 - 12 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female Chinese patients, aged 1 to ≤12 years at ICF signature with radiographic evidence of rickets
•2. Diagnosis of XLH supported by either of the following:
•* Confirmed PHEX mutation (prior to the study with historic record) in the patient or a directly related family member with approximate X linked inheritance
•* Serum intact FGF23 level ≥30 pg/mL by Kainos assay at Screening
•3. Able to receive conventional therapy (oral phosphate and pharmacologic vitamin D)
•4. Biochemical findings associated with XLH: Serum phosphorus \<3.0 mg/dL (0.97 mmol/L). Serum phosphorus may be re tested (once only) at least 7 days after discontinuation of therapy, if applicable (\[see Section 3.1\])
•5. Serum creatinine within age-adjusted normal range (based on overnight fasting \[minimum 4 hours\] values collected at Screening)
•6. Serum 25(OH)D above or equal to the lower limit of normal (≥16 ng/mL) at Screening. If 25(OH)D levels are below the normal range, 25(OH)D supplementation will be prescribed. Assuming a patient meets all other eligibility requirements, the patient may be re tested for serum 25(OH)D after a minimum of 7 days of treatment
•7. Willing to provide access to prior medical records for the collection of historical growth and radiographic data and disease history
•8. Written or verbal assent (as appropriate for the patient and region) by the patient and written informed consent by legally authorized representatives provided after the nature of the study has been explained, and prior to any research-related procedures
+2 more criteria

Exclusion Criteria

•1. Positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) antibody at Screening
•2. Tanner stage 4 or higher through physical examination
•3. Height percentile \>50% based on country specific norms
•4. Use of a pharmacologic vitamin D, its metabolites, or analogs, oral phosphate for treatment of XLH, aluminum hydroxide antacids, acetazolamide, thiazide diuretics, and/or systemic corticosteroids within 7 days prior to Week -14
•5. Current or prior use of leuprorelin, triptorelin, goserelin, or other drugs known to delay puberty
•6. Use of growth hormone therapy within 12 months before ICF signature
•7. Have uncontrolled diabetes mellitus, defined as glycated hemoglobin (HbA1c) \>8.5% at Screening
•8. Presence of nephrocalcinosis on renal ultrasound Grade 4 based on the following scale:
•9. Planned or recommended orthopedic surgery (implantation or removal), including staples, 8 plates, or osteotomy, within the first 40 weeks of the study
•10. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age adjusted normal limits (based on overnight fasting \[minimum 4 hours\] values collected at the Screening)
+9 more criteria

Locations (5)

Peking Union Medical College Hospital

Beijing, China

Guangzhou Women and Children Medical Center

Guangzhou, China

Shanghai 6th Hospital

Shanghai, China

Shanghai Xinhua Hopsital

Shanghai, China

Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology

Wuhan, China

Key Dates

Start Date

November 1, 2021

Primary Completion Date

December 18, 2023

Completion Date

December 18, 2023

Last Updated

July 9, 2024

Enrollment

ACTUAL participants

Conditions

X-linked Hypophosphatemia (XLH)

Interventions

KRN23

DRUG

Prevention of Spontaneous Dental Abscesses in Children With X-linked Hypophosphatemia : a RCT

NCT04872907

X-linked Hypophosphatemia (XLH)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 9, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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