CompletedPhase 3

Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

United States14 participantsStarted 2015-12-23

Plain-language summary

The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).

Who can participate

Age range18 Years – 65 Years

SexALL

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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 based on overnight fasting (min. 8 hours):
✓. Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥ 4 on the Brief Pain Inventory question 3 (BPI-Q3, Worst Pain) at Screening. (Skeletal pain that, in the opinion of the investigator, is attributed solely to causes other than XLH/osteomalacia-for example, 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)
✓. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] equation) or estimated glomerular filtration rate (eGFR) eGFR of 45 to \<60 mL/min at Screening with confirmation that the renal insufficiency is not due to nephrocalcinosis
✓. Provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures. If the subject in a minor, provide written assent 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
✓. Females of child-bearing potential must have a negative urine pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not to be of childbearing potential include those who have been in menopause for at least two years prior to Screening, or have had tubal ligation at least one year prior to Screening, or have had a total hysterectomy or bilateral salpingo-oophorectomy.

Exclusion criteria

✕. Use of any pharmacologic vitamin D metabolite or analog (e.g. calcitriol, doxercalciferol, and paricalcitol) within the 2 years before Screening
✕. Use of oral phosphate within 2 years before Screening
✕. Use of aluminum hydroxide antacids, acetazolamides, and thiazides within 7 days prior to Screening
✕. Use of bisphosphonates in the 2 years prior to Screening
✕. Use of denosumab in the 6 months prior to Screening
✕. Use of teriparatide in the 2 months prior to Screening
✕. Chronic use of systemic corticosteroids defined as more than 10 days in the 2 months prior to Screening
✕. Corrected serum calcium level ≥ 10.8 mg/dL (2.7 mmol/L) at Screening

What they're measuring

Percent Change From Baseline in OV/BV at Week 48

Timeframe: Baseline, 48 weeks

Trial details

NCT IDNCT02537431

SponsorKyowa Kirin, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-08-30

Results submitted2018-08-27

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