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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
X-linked Hypophosphatemia Drug: Calcitriol Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
Actual Study Start Date : January 1, 2017
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rickets Vitamin D

Arms and Interventions
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Arm Intervention/treatment
Experimental: High-dose
Receiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.
 Drug: Calcitriol
Other Name: Elementary phosphorus
Experimental: Low-dose
Receiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus.
 Drug: Calcitriol
Other Name: Elementary phosphorus


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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.

  9. 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

  10. 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

  11. 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


Eligibility Criteria
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Ages Eligible for Study:   1 Year to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03820518


Contacts
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Contact: Weibo Xia, MD +86 13501002126 xiaweibo8301@163.com

Locations
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China, Beijing
Department of Endocrinology, Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Weibo Xia, MD    +86 13501002126    xiaweibo8301@163.com   
Sponsors and Collaborators
Peking Union Medical College Hospital
More Information
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Responsible Party: Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03820518    
Other Study ID Numbers: JS-1824
First Posted: January 29, 2019    Key Record Dates
Last Update Posted: January 30, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Familial Hypophosphatemic Rickets
Hypophosphatemia
Phosphorus Metabolism Disorders
Metabolic Diseases
Rickets, Hypophosphatemic
Rickets
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Calcitriol
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Calcium Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasoconstrictor Agents
Vitamins