NCT03820518 - Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia | Crick

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

100

Conditions

X-linked Hypophosphatemia

Interventions

CalcitriolDRUG

Eligibility

Sex: ALLMin age: 1 YearMax age: 12 Years

Medical Language ↔ Plain English

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

Locations (1)

Department of Endocrinology, Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score

RSS range from 0 to 10, and higher RSS represent severer rickets. It is consisted of score of worst wrist (0-4) and worst knee (0-6). WRIST-score both radius and ulna separately-2 bones × 2 points = 4 points possible KNEE-score both femur and tibia separately Multiply the grade in A by the multiplier in B for each bone, then add femur and tibia scores together A: Grade 1 2 3 B: Multiplier Portion of growth plate affected 0.5 ≤ 1 condyle or plateau 1. 2 condyles or plateaus 2. bones × 1 point × 3 points = 6 points possible Total: 10 points possible Reference:Thacher, T. Radiographic scoring method for the assessment of the severity of nutritional rickets\[J\]. Journal of Tropical Pediatrics, 2000, 46(3):132-139.

Time frame: Baseline, Month 12, 24

Secondary Outcomes

Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score

RSS range from 0 to 10, and higher RSS represent severer rickets. It is consisted of score of worst wrist (0-4) and worst knee (0-6). WRIST-score both radius and ulna separately-2 bones × 2 points = 4 points possible KNEE-score both femur and tibia separately Multiply the grade in A by the multiplier in B for each bone, then add femur and tibia scores together A: Grade 1 2 3 B: Multiplier Portion of growth plate affected 0.5 ≤ 1 condyle or plateau 1. 2 condyles or plateaus 2. bones × 1 point × 3 points = 6 points possible Total: 10 points possible Reference:Thacher, T. Radiographic scoring method for the assessment of the severity of nutritional rickets\[J\]. Journal of Tropical Pediatrics, 2000, 46(3):132-139.

Time frame: Baseline, Month 12, 24

Changes From Baseline to Post-treatment in Growth Velocity

Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score

Time frame: Baseline, Month 12, 24

Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels

Central Contacts

Weibo Xia, MD

CONTACT

+86 13501002126 xiaweibo8301@163.com