Status:

UNKNOWN

Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia

Lead Sponsor:

Peking Union Medical College Hospital

Conditions:

X-linked Hypophosphatemia

Eligibility:

All Genders

1-12 years

Phase:

PHASE4

Brief Summary

X-linked hypophosphatemia (XLH) is the most common form of heritable rickets. Current treatments include active vitamin D metabolites (e.g. calcitriol) and phosphate salts. There is no consistent weig...

Eligibility Criteria

Inclusion

  • Male or female, aged 1-12 years, inclusive
  • Diagnosis of XLH by clinical features: serum phosphorus level \< 2.5 mg/dl; ALP?; RSS total score ≥2; bowed legs; short stature; family history with appropriate X-linked inheritance
  • Meet at least one of the following: confirmed Phosphate regulating gene with homology to endopeptidases located on the X chromosome (PHEX) mutation in the participant, or serum FGF23 level \>30 pg/ml (Kainos assay)
  • Willing to participate the study, and provide an informed consent
  • Able to complete all aspects of study and adhere to the visit schedule

Exclusion

  • Use of growth hormone within 12 months before first visit
  • Height \>50 percentile for age and sex specific data
  • Presence of nephrocalcinosis or nephrolithiasis
  • Serum intact parathyroid hormone level\>170 pg/ml
  • Plan to receive orthopaedic surgery in 12 months
  • Poor compliance
  • Use of gonadotropin-releasing hormone therapy right now
  • Use of aluminium hydroxide, steroid, acetazolamide or thiazide drugs within 7 days before first visit
  • Not be fit to participant in the study, by the judgement of investigators

Key Trial Info

Start Date :

January 1 2017

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 31 2022

Estimated Enrollment :

100 Patients enrolled

Trial Details

Trial ID

NCT03820518

Start Date

January 1 2017

End Date

December 31 2022

Last Update

January 30 2019

Active Locations (1)

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1

Department of Endocrinology, Peking Union Medical College Hospital

Beijing, Beijing Municipality, China, 100730