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

NCT ID: NCT03820518

Last Updated: 2019-01-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-01

Study Completion Date

2022-12-31

Brief Summary

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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

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Conditions

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X-linked Hypophosphatemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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High-dose

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

Group Type EXPERIMENTAL

Calcitriol

Intervention Type DRUG

Low-dose

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

Group Type EXPERIMENTAL

Calcitriol

Intervention Type DRUG

Interventions

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Calcitriol

Intervention Type DRUG

Other Intervention Names

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Elementary phosphorus

Eligibility Criteria

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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
Minimum Eligible Age

1 Year

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Peking Union Medical College Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Department of Endocrinology, Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Weibo Xia, MD

Role: CONTACT

+86 13501002126

Facility Contacts

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Weibo Xia, MD

Role: primary

+86 13501002126

Other Identifiers

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JS-1824

Identifier Type: -

Identifier Source: org_study_id

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