X-linked HypophosphatemiaPhase 4NCT03820518
Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
A Phase 4 study testing Calcitriol in people with X-linked Hypophosphatemia, run by Peking Union Medical College Hospital at 1 site in China.
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. Read more about this study
- Who is it for?
- Ages 1 year to 12 years
- Conditions the study is for
- X-linked Hypophosphatemia
- What is being tested?
- Calcitriol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2017to 31 Dec 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Endocrinology, Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Weibo Xia, MD, +86 13501002126, xiaweibo8301@163.com
About this study
Official title: Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
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.
- Conditions
- X-linked Hypophosphatemia
- Being tested
- Calcitriol
- Medical subject headings
- Familial Hypophosphatemic Rickets
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CalcitriolDrug
Also called Elementary phosphorus
| Study group | Role | What they receive |
|---|---|---|
| High-dose | Experimental | Receiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. Receives: Drug: Calcitriol |
| Low-dose | Experimental | Receiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. Receives: Drug: Calcitriol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Month 12, 24
Secondary outcomes (11)
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.
Measured: Baseline, Month 12, 24
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
Measured: Baseline, Month 12, 24
Changes From Baseline to Post-treatment in Serum Total Alkaline Phosphatase (TALP) Levels
Changes From Baseline to Post-treatment in serum ALP
Measured: Baseline, Month 3, 6, 12, 18, 24
Changes From Baseline to Post-treatment in serum Carboxy-terminal Collagen Crosslinks (CTX) Levels
Changes From Baseline to Post-treatment in serum CTX
Measured: Baseline, Month 3, 6, 12, 18, 24
Changes From Baseline to Post-treatment in Serum Phosphorus Levels
Changes From Baseline to Post-treatment in Serum Phosphorus Levels
Measured: Baseline, Month 3, 6, 12, 18, 24
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
Measured: Baseline, Month 3, 6, 12, 18, 24
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
Measured: Baseline, Month 3, 6, 12, 18, 24
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.
Measured: Baseline, Month 3, 6, 12, 18, 24
Changes From Baseline to Post-treatment in Serum Osteocalcin Levels
Changes From Baseline to Post-treatment in Serum Osteocalcin Levels
Measured: Baseline, Month 3, 6, 12, 18, 24
Changes From Baseline to Post-treatment in Height
Changes From Baseline to Post-treatment in Height
Measured: 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
Changes From Baseline to Post-treatment in Quality of Life as Measured by Patient-reported Outcomes Measurement Information System
Measured: Baseline, Month 3, 6, 12, 18, 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2019
- Study start1 Jan 2017
- Primary completion31 Dec 2021 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-linked HypophosphatemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03820518
- Sponsor's protocol ID
- JS-1824
- Status last verified
- Jan 2019
- First submitted
- 25 Jan 2019
- Last updated
- 30 Jan 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparing the Effectiveness of High or Low Dose of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
- ClinicalTrials.gov ID
- NCT03820518
- Acronym / sponsor ID
- JS-1824
- Conditions
- X-linked Hypophosphatemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 29 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| High-dose | Experimental | Drug: CalcitriolReceiving 60 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. |
| Low-dose | Experimental | Drug: CalcitriolReceiving 20 ng/kg/day of calcitriol and 30 mg/kg/day of elemental phosphorus. |
Who may qualify
- Ages
- 1 year to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CalcitriolAlso: Elementary phosphorus | Drug | High-dose, Low-dose |
Primary outcome
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.
11 secondary outcomes
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 Growth Velocity
Time frame: Baseline, Month 12, 24
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 Carboxy-terminal Collagen Crosslinks (CTX) Levels
Time frame: Baseline, Month 3, 6, 12, 18, 24
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 Severity of Dental Abscess
Time frame: Baseline, Month 3, 6, 12, 18, 24
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 Severity of Leg Deformities
Time frame: Baseline, Month 3, 6, 12, 18, 24
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 Height
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
Time frame: Baseline, Month 3, 6, 12, 18, 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Calcitriol in people with X-linked Hypophosphatemia, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 30 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Weibo Xia, MD (+86 13501002126, xiaweibo8301@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2019.