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

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ClinicalTrials.gov Identifier: NCT03820518
Recruitment Status : Recruiting
First Posted : January 29, 2019
Last Update Posted : January 30, 2019
Sponsor:
Information provided by (Responsible Party):
Peking Union Medical College Hospital

Tracking Information
First Submitted Date  ICMJE January 25, 2019
First Posted Date  ICMJE January 29, 2019
Last Update Posted Date January 30, 2019
Actual Study Start Date  ICMJE January 1, 2017
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 28, 2019)
Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score [ Time Frame: Baseline, Month 12, 24 ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 25, 2019)
Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score [ Time Frame: Baseline, Month 12, 24 ]
Change From Baseline to Post-treatment in Severity of Rickets as Measured by Rickets Severity Score (RSS) Total Score
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 28, 2019)
  • Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score [ Time Frame: Baseline, Month 12, 24 ]
    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.
  • Changes From Baseline to Post-treatment in Growth Velocity [ Time Frame: Baseline, Month 12, 24 ]
    Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score
  • Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in serum ALP
  • Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in serum CTX
  • Changes From Baseline to Post-treatment in Serum Phosphorus Levels [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Serum Phosphorus Levels
  • Changes From Baseline to Post-treatment in Severity of Dental Abscess [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    The frequency and number of dental abscess will be collected by self-report and examed by investigators
  • Changes From Baseline to Post-treatment in Severity of Bone Pain as Measured by Visual Analog Pain Scales [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Visual Analog Pain Scales (VAS) range from 0-10, and higher score represents severer pain
  • Changes From Baseline to Post-treatment in Severity of Leg Deformities [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    The longest distance between two sides of thighs, knees, shanks and ankles are measured to evaluate the severity of leg deformities.
  • Changes From Baseline to Post-treatment in Serum Osteocalcin Levels [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Serum Osteocalcin Levels
  • Changes From Baseline to Post-treatment in Height [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Height
  • Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System
Original Secondary Outcome Measures  ICMJE
 (submitted: January 25, 2019)
  • 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 Severity of Rickets as Measured by RSS Wrist and Knee Score
  • Changes From Baseline to Post-treatment in Growth Velocity [ Time Frame: Baseline, Month 12, 24 ]
    Changes From Baseline to Post-treatment in Severity of Rickets as Measured by RSS Wrist and Knee Score
  • Changes From Baseline to Post-treatment in Alkaline Phosphatase [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Alkaline Phosphatase
  • Changes From Baseline to Post-treatment in Carboxy-terminal Collagen Crosslinks [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Carboxy-terminal Collagen Crosslinks
  • Changes From Baseline to Post-treatment in Serum Phosphorus Levels [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Carboxy-terminal Collagen Crosslinks
  • Changes From Baseline to Post-treatment in Severity of Dental Abscess [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Carboxy-terminal Collagen Crosslinks
  • Changes From Baseline to Post-treatment in Severity of Bone Pain as Measured by Visual Analog Pain Scales [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Carboxy-terminal Collagen Crosslinks
  • Changes From Baseline to Post-treatment in Severity of Leg Deformities [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    The longest distance between two sides of thighs, knees, shanks and ankles are measured to evaluate the severity of leg deformities.
  • Changes From Baseline to Post-treatment in Osteocalcin [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Osteocalcin
  • Changes From Baseline to Post-treatment in Height Z Score [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Height Z Score
  • Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System [ Time Frame: Baseline, Month 3, 6, 12, 18, 24 ]
    Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
Official Title  ICMJE Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE X-linked Hypophosphatemia
Intervention  ICMJE Drug: Calcitriol
Other Name: Elementary phosphorus
Study Arms  ICMJE
  • Experimental: High-dose
    Receiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.
    Intervention: Drug: Calcitriol
  • Experimental: Low-dose
    Receiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.
    Intervention: Drug: Calcitriol
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 25, 2019)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2022
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Weibo Xia, MD +86 13501002126 xiaweibo8301@163.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03820518
Other Study ID Numbers  ICMJE JS-1824
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Peking Union Medical College Hospital
Study Sponsor  ICMJE Peking Union Medical College Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Peking Union Medical College Hospital
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP