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

NCT03820518 | Unknown Phase 4

• 1 other identifier: JS-1824
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

X-linked Hypophosphatemia

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Month 12, 24

Secondary Outcomes (11)

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

  Baseline, Month 12, 24

• Changes From Baseline to Post-treatment in Growth Velocity

  Baseline, Month 12, 24

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

  Baseline, Month 3, 6, 12, 18, 24

• Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels

  Baseline, Month 3, 6, 12, 18, 24

• Changes From Baseline to Post-treatment in Serum Phosphorus Levels

  Baseline, Month 3, 6, 12, 18, 24

Study Arms (2)

High-dose

EXPERIMENTAL

Receiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.

Drug: Calcitriol

Low-dose

EXPERIMENTAL

Receiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.

Drug: Calcitriol

Interventions

Calcitriol DRUG

Also known as: Elementary phosphorus

High-dose; Low-dose

Eligibility Criteria

• Age: 1 Year - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Department of Endocrinology, Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Familial Hypophosphatemic Rickets

Interventions

Calcitriol

Condition Hierarchy (Ancestors)

Rickets, Hypophosphatemic; Rickets; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Hypophosphatemia, Familial; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Hypophosphatemia; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Intervention Hierarchy (Ancestors)

Dihydroxycholecalciferols; Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Central Study Contacts

Weibo Xia, MD

CONTACT

+86 13501002126; xiaweibo8301@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2019
• First Posted: January 29, 2019
• Study Start: January 1, 2017
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2022
• Last Updated: January 30, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)