Clinical trial NCT03820518: Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
Official title: Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: X-linked Hypophosphatemia
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2019-01-29
Enrollment: 100
Sex: ALL
Minimum age (years): 1
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 weight-based dosing of calcitriol and phosphate now. The primary objective of this study is to establish the efficacy of different dose of calcitriol combined with neutral phosphate in children with XLH.


Eligibility criteria: Inclusion Criteria:

* 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 Criteria:

* 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

Primary outcomes:
measure: Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score
timeFrame: Baseline, Month 12, 24
Secondary outcomes:
measure: Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score
timeFrame: Baseline, Month 12, 24
measure: Changes From Baseline to Post-treatment in Growth Velocity
timeFrame: Baseline, Month 12, 24
measure: Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Serum Phosphorus Levels
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Severity of Dental Abscess
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Severity of Bone Pain as Measured by Visual Analog Pain Scales
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Severity of Leg Deformities
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Serum Osteocalcin Levels
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Height
timeFrame: Baseline, Month 3, 6, 12, 18, 24
measure: Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System
timeFrame: Baseline, Month 3, 6, 12, 18, 24
Other outcomes:
Locations:
city: Beijing
country: China
facility: Department of Endocrinology, Peking Union Medical College Hospital
state: Beijing
Study officials:
References: