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

Back to results

Primary Purpose

Status

Unknown status

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Calcitriol

About this trial

This is an interventional treatment trial for X-linked Hypophosphatemia

Eligibility Criteria

1 Year - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Department of Endocrinology, Peking Union Medical College HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

High-dose

Low-dose

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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

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

Changes From Baseline to Post-treatment in serum ALP

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

Changes From Baseline to Post-treatment in serum CTX

Changes From Baseline to Post-treatment in Serum Phosphorus Levels

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

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

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.

Changes From Baseline to Post-treatment in Serum Osteocalcin Levels

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

Full Information

NCT ID

NCT03820518

First Posted

January 25, 2019

Last Updated

January 28, 2019

Sponsor

Peking Union Medical College Hospital

1. Study Identification

Unique Protocol Identification Number

NCT03820518

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2019

Overall Recruitment Status

Unknown status

Study Start Date

January 1, 2017 (Actual)

Primary Completion Date

December 31, 2021 (Anticipated)

Study Completion Date

December 31, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Peking Union Medical College Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

No

Studies a U.S. FDA-regulated Device Product

No

Data Monitoring Committee

No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

X-linked Hypophosphatemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

High-dose

Arm Type

Experimental

Arm Description

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

Arm Title

Low-dose

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Calcitriol

Other Intervention Name(s)

Elementary phosphorus

Primary Outcome Measure Information:

Title

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

Description

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.

Time Frame

Baseline, Month 12, 24

Secondary Outcome Measure Information:

Title

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

Description

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.

Time Frame

Baseline, Month 12, 24

Title

Changes From Baseline to Post-treatment in Growth Velocity

Description

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

Time Frame

Baseline, Month 12, 24

Title

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

Description

Changes From Baseline to Post-treatment in serum ALP

Time Frame