CompletedPhase 3

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

United States134 participantsStarted 2015-10-22

Plain-language summary

The primary efficacy objective of this study is to establish the effect of burosumab treatment compared with placebo on increasing serum phosphorus levels in adults with XLH.

Who can participate

Age range18 Years – 65 Years

SexALL

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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 18 - 65 years, inclusive
✓. Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs) and at least ONE of the following at Screening:
✓. Biochemical findings consistent with XLH at Screening Visit 2 following overnight fasting (min. 8 hours):
✓. Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥ 4 on Brief Pain Inventory (BPI) Questions 3 (Worst Pain) at Screening Visit 1. (Skeletal pain that, in the opinion of the investigator, is attributed solely to causes other than XLH/osteomalacia-for example, back 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)
✓. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] equation) or eGFR of 45 60 mL/min at Screening Visit 2 with confirmation that the renal insufficiency is not due to nephrocalcinosis
✓. If taking chronic pain medications (including narcotic pain medications/opioids), must be on a stable regimen for at least 21 days prior to Screening Visit 1, and be willing to maintain medications at the same stable dose(s) and schedule throughout the Placebo-controlled Treatment Period of the study. The dose must not exceed 60 mg oral morphine equivalents/day
✓. Provide written informed consent or if a minor, provide written consent and have a legally authorized representative willing and able to provide written informed consent, 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 metabolite or analog (calcitriol, doxercalciferol, and paricalcitol) within 14 days prior to Screening Visit 2
✕. Use of oral phosphate within 14 days prior to Screening Visit 2
✕. Use of aluminum hydroxide antacids, acetazolamides, and thiazides within 7 days prior to Screening Visit 2
✕. Chronic use of systemic corticosteroids defined as more than 10 days in the previous 2 months
✕. Corrected serum calcium level ≥ 10.8 mg/dL (2.7 mmol/L) at Screening Visit 2
✕. Serum intact parathyroid hormone (iPTH) ≥ 2.5 upper limit of normal (ULN) at Screening Visit 1
✕. Use of medication to suppress parathyroid hormone (PTH) (cinacalcet for example) within 60 days prior to Screening Visit 1
✕. Use of oral bisphosphonates for 6 months or more in the 2 years prior to Screening Visit 1

What they're measuring

Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24

Timeframe: Baseline through Week 24

Trial details

NCT IDNCT02526160

SponsorKyowa Kirin, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2016-12-22

Results submitted2020-12-17

View on ClinicalTrials.gov More X-linked Hypophosphatemia trials