CompletedPhase 4

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With TIO

China9 participantsStarted 2022-09-07

Plain-language summary

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

Age range18 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.

✓. Have a clinical diagnosis of TIO based on evidence of excessive FGF23 that is not amenable to cure by surgical excision of the offending tumor (documented by Investigator)
✓. Male or female Chinese patients aged ≥18 years at the time of signing the informed consent form
✓. Have a fasting serum phosphorus level \< 2.5 mg/dL (0.81 mmol/L) at Screening
✓. Have a serum iFGF23 level ≥ 100 pg/mL by Kainos assay at Screening
✓. Have a TmP/GFR \< 2.5 mg/dL at Screening
✓. Have an estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m2(using CKD-EPI formula) at Screening. Subjects with an eGFR ≥ 30 but \< 60 mL/min at screening will be considered eligible so long as in the opinion of the Investigator the decline in renal function is not related to nephrocalcinosis
✓. Have a corrected serum calcium level \< 10.8 mg/dL (2.69 mmol/L) at Screening (Corrected serum calcium = serum calcium in mg/dL + 0.8 × \[4 - serum albumin in g/dL\])
✓. Have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study (female patients of child-bearing potential only)

✕. Use of the following drugs within 14 days prior to screening: pharmacologic vitamin D metabolites or analogs, or drugs for treating TIO including oral phosphate, aluminum hydroxide antacids, acetazolamide, or thiazide diuretics
✕. Medication to suppress parathyroid hormone (PTH) (e.g., cinacalcet hydrochloride) within 60 days prior to screening
✕. Blood or blood product transfusion within 60 days prior to screening
✕. History of malignancy within 5 years of study entry with the exception of PMT-MCT (phosphaturic mesenchymal tumors of the mixed connective tissue type)
✕. Positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) antibody at Screening, or prior history of positive test
✕. Predisposition to infection, or history of recurrent infection or known immunodeficiency
✕. Pregnant or breastfeeding at screening or intention to become pregnant during the study; for male subjects, the partner's intention to become pregnant during the study
✕. Use of an investigational product (IP) or device within 4 months prior to screening, or planning to receive other IP before completing all assessments in this study.

Change from Baseline in mean serum phosphorus level at the end of the dosing cycle.

Timeframe: Week 20, 24, 28, 32, 36, 40, 44 and 48

NCT IDNCT05357573

SponsorKyowa Kirin Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-11-22

Who can participate

Age range18 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.

✓. Have a clinical diagnosis of TIO based on evidence of excessive FGF23 that is not amenable to cure by surgical excision of the offending tumor (documented by Investigator)
✓. Male or female Chinese patients aged ≥18 years at the time of signing the informed consent form
✓. Have a fasting serum phosphorus level \< 2.5 mg/dL (0.81 mmol/L) at Screening
✓. Have a serum iFGF23 level ≥ 100 pg/mL by Kainos assay at Screening
✓. Have a TmP/GFR \< 2.5 mg/dL at Screening
✓. Have an estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m2(using CKD-EPI formula) at Screening. Subjects with an eGFR ≥ 30 but \< 60 mL/min at screening will be considered eligible so long as in the opinion of the Investigator the decline in renal function is not related to nephrocalcinosis
✓. Have a corrected serum calcium level \< 10.8 mg/dL (2.69 mmol/L) at Screening (Corrected serum calcium = serum calcium in mg/dL + 0.8 × \[4 - serum albumin in g/dL\])
✓. Have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study (female patients of child-bearing potential only)

✕. Use of the following drugs within 14 days prior to screening: pharmacologic vitamin D metabolites or analogs, or drugs for treating TIO including oral phosphate, aluminum hydroxide antacids, acetazolamide, or thiazide diuretics
✕. Medication to suppress parathyroid hormone (PTH) (e.g., cinacalcet hydrochloride) within 60 days prior to screening
✕. Blood or blood product transfusion within 60 days prior to screening
✕. History of malignancy within 5 years of study entry with the exception of PMT-MCT (phosphaturic mesenchymal tumors of the mixed connective tissue type)
✕. Positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) antibody at Screening, or prior history of positive test
✕. Predisposition to infection, or history of recurrent infection or known immunodeficiency
✕. Pregnant or breastfeeding at screening or intention to become pregnant during the study; for male subjects, the partner's intention to become pregnant during the study
✕. Use of an investigational product (IP) or device within 4 months prior to screening, or planning to receive other IP before completing all assessments in this study.