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

X-linked Hypophosphatemia Clinical Trial

Official title:

Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03820518
• Other study ID #: JS-1824
• Status: Recruiting
• Phase: Phase 4
• Start date: January 1, 2017
• Est. completion date: December 31, 2022

Study information

• Verified date: January 2019
• Source: Peking Union Medical College Hospital
• Contact: Weibo Xia, MD
• Phone: +86 13501002126
• Email: xiaweibo8301[@]163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 31, 2022
• Est. primary completion date: December 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• X-linked Hypophosphatemia

Intervention

Drug:
• Calcitriol

Locations

Country	Name	City	State
China	Department of Endocrinology, Peking Union Medical College Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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; 2 condyles or plateaus; 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	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; 2 condyles or plateaus; 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	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	Baseline, Month 12, 24
Secondary	Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels	Changes From Baseline to Post-treatment in serum ALP	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels	Changes From Baseline to Post-treatment in serum CTX	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Serum Phosphorus Levels	Changes From Baseline to Post-treatment in Serum Phosphorus Levels	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Severity of Dental Abscess	The frequency and number of dental abscess will be collected by self-report and examed by investigators	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Severity of Bone Pain as Measured by Visual Analog Pain Scales	Visual Analog Pain Scales (VAS) range from 0-10, and higher score represents severer pain	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Severity of Leg Deformities	The longest distance between two sides of thighs, knees, shanks and ankles are measured to evaluate the severity of leg deformities.	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Serum Osteocalcin Levels	Changes From Baseline to Post-treatment in Serum Osteocalcin Levels	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Height	Changes From Baseline to Post-treatment in Height	Baseline, Month 3, 6, 12, 18, 24
Secondary	Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System	Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System	Baseline, Month 3, 6, 12, 18, 24