CompletedPhase 4

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

China18 participantsStarted 2021-09-28

Plain-language summary

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

Who can participate

Age range18 Years – 65 Years

SexALL

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Inclusion criteria

✓. Male or female Chinese patients, aged 18 to 65 years (inclusive) at the time of signing the ICF
✓. Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs) and at least either of the following at screening:
✓. Biochemical findings consistent with XLH following overnight fasting (≥8 hours) at Screening:
✓. Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥4 on the BPI Worst Pain question at Screening (Skeletal pain that, in the opinion of the investigator or subinvestigator, is attributed solely to causes other than XLH/osteomalacia \[e.g., back pain or joint pain in the presence of severe osteoarthritis by radiograph in that anatomical location\] in the absence of any skeletal pain likely attributed to XLH/osteomalacia should not be considered for eligibility)
✓. Patients who are taking chronic pain medications (including narcotic pain medications/opioids) must be on a stable regimen for at least 21 days before signing the ICF and be willing to maintain the medications at the same stable dose(s) and schedule throughout the study. The dose must not exceed 60 mg oral morphine equivalents/day
✓. Able to receive conventional therapy (oral phosphate and pharmacologic vitamin D \[or metabolites/analogs\])
✓. Written informed consent provided after the nature of the study has been explained and prior to any research related procedures
✓. Willing to provide access to prior medical records for the collection of biochemical and radiographic data and disease history

Exclusion criteria

✕. Use of a pharmacologic vitamin D, its metabolites, or analogs, and oral phosphate for treatment of XLH within 14 days prior to Screening.
✕. Use of aluminum hydroxide antacids, acetazolamide, thiazide diuretics and/or systemic corticosteroids within 14 days prior to Week -14
✕. Corrected serum calcium level ≥10.8 mg/dL (2.69 mmol/L) at Screening
✕. Plasma iPTH ≥2.5 times the upper limit of normal at Screening
✕. Uncontrolled diabetes mellitus, defined as HbA1c \>7.5% at Screening
✕. Use of medication to suppress PTH (e.g., Sensipar®, cinacalcet, calcimimetics) within 60 days before signing the ICF
✕. Use of oral bisphosphonates in the 2 years before signing the ICF
✕. Planned or recommended orthopedic surgery within the clinical trial period

What they're measuring

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

Timeframe: Weeks 4, 8, 12, 16, 20, 24, 36, and 48

Trial details

NCT IDNCT04842019

SponsorKyowa Kirin Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-08-17

View on ClinicalTrials.gov More X-linked Hypophosphatemia (XLH) trials