CompletedPhase 4

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

China28 participantsStarted 2021-11-01

Plain-language summary

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

Age range1 Year – 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.

✓. Male or female Chinese patients, aged 1 to ≤12 years at ICF signature with radiographic evidence of rickets
✓. Diagnosis of XLH supported by either of the following:
✓. Able to receive conventional therapy (oral phosphate and pharmacologic vitamin D)
✓. 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 (\[see Section 3.1\])
✓. Serum creatinine within age-adjusted normal range (based on overnight fasting \[minimum 4 hours\] values collected at Screening)
✓. 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
✓. Willing to provide access to prior medical records for the collection of historical growth and radiographic data and disease history
✓. 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

✕. Positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) antibody at Screening
✕. Tanner stage 4 or higher through physical examination
✕. Height percentile \>50% based on country specific norms
✕. 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
✕. Current or prior use of leuprorelin, triptorelin, goserelin, or other drugs known to delay puberty
✕. Use of growth hormone therapy within 12 months before ICF signature
✕. Have uncontrolled diabetes mellitus, defined as glycated hemoglobin (HbA1c) \>8.5% at Screening
✕. Presence of nephrocalcinosis on renal ultrasound Grade 4 based on the following scale:

Change from Baseline (CFB) in mean serum phosphorus level at the end of the dose cycle

Timeframe: Weeks 2, 4, 8, 12, 16, 24, 32, 40, 52, and 64

NCT IDNCT04842032

SponsorKyowa Kirin Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-12-18

Who can participate

Age range1 Year – 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.

✓. Male or female Chinese patients, aged 1 to ≤12 years at ICF signature with radiographic evidence of rickets
✓. Diagnosis of XLH supported by either of the following:
✓. Able to receive conventional therapy (oral phosphate and pharmacologic vitamin D)
✓. 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 (\[see Section 3.1\])
✓. Serum creatinine within age-adjusted normal range (based on overnight fasting \[minimum 4 hours\] values collected at Screening)
✓. 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
✓. Willing to provide access to prior medical records for the collection of historical growth and radiographic data and disease history
✓. 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

✕. Positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) antibody at Screening
✕. Tanner stage 4 or higher through physical examination
✕. Height percentile \>50% based on country specific norms
✕. 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
✕. Current or prior use of leuprorelin, triptorelin, goserelin, or other drugs known to delay puberty
✕. Use of growth hormone therapy within 12 months before ICF signature
✕. Have uncontrolled diabetes mellitus, defined as glycated hemoglobin (HbA1c) \>8.5% at Screening
✕. Presence of nephrocalcinosis on renal ultrasound Grade 4 based on the following scale: