- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03748966
Calcitriol Monotherapy for X-Linked Hypophosphatemia
March 11, 2024 updated by: Eva Liu, Massachusetts General Hospital
Calcitriol Monotherapy for X-Linked Hypophosphatemia: Effects on Mineral Ions, Growth and Skeletal Parameters
Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose will be escalated during the first 3 months of therapy.
The investigators hypothesize that treatment of adults and children with XLH alone will improve serum phosphate levels and skeletal mineralization without causing an increase in kidney calcifications.
The study will also examine if calcitriol therapy will improve growth in children.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
20
Phase
- Early Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Eva S Liu, MD
- Phone Number: 16175255412
- Email: esliu@bwh.harvard.edu
Study Contact Backup
- Name: Marie Demay, MD
- Phone Number: 16177263273
- Email: demay@helix.mgh.harvard.edu
Study Locations
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02114
- Recruiting
- Massachusetts General Hospital
-
Contact:
- Eva Liu, MD
- Phone Number: 617-525-5412
- Email: esliu@bwh.harvard.edu
-
Contact:
- Marie Demay, MD
- Phone Number: 6177263273
- Email: demay@helix.mgh.harvard.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years to 70 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Clinical diagnosis of XLH with family history excluding male-to-male transmission, or positive genotype for PHEX mutation
- Serum PTH levels less than 1.5x the upper limit of normal
- Serum calcium levels less than 10.0 mg/dl
- eGFR >= 60 mL/min/1.73m2
- 25(OH) vitamin D level >= 20 ng/dL
Exclusion Criteria:
- Known allergy to calcitriol
- Pregnancy or breast feeding
- Use of skeletally active agents such as bisphosphonates, teriparatide, SERMS, hormone replacement therapy and progesterone-only contraceptive agents (combination oral contraceptive use in premenopausal women is not an exclusion criterion).
- Unwilling or unable to stop therapy with calcitriol and phosphate therapy for two weeks prior to study
- Therapy with cinacalcet within the past two weeks
- Current use of growth hormone therapy
- Use of diuretics or medications that alter renal handling of mineral ions.
- Use of glucocorticoids for more than 14 days in the past 12 months with the exception of inhaled agents.
- History of malignancy except basal and squamous cell carcinoma of the skin.
- Significant history of psychiatric disease per DSM-5.
- Substance use disorder per DSM-5.
- Significant cardiopulmonary disease (unstable CAD or stage D ACC/AHA heart failure).
- Absence of laboratory values for serum calcium, phosphate and creatinine in the 24 months prior to enrollment.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Adults with XLH
Adults with X-linked hypophosphatemia will be treated with optimized doses of calcitriol (without phosphate supplementation) for one year
|
Adults and children (age 3-17) with X-linked hypophosphatemia will be treated with calcitriol therapy without phosphate supplementation.
Doses of calcitriol will be escalated and optimized in the first three months of the study.
Calcitriol is an oral medication taken once a day.
Other Names:
|
Experimental: Children with XLH
Children (age 3-17) with X-linked hypophosphatemia will be treated with optimized doses of calcitriol (without phosphate supplementation) for one year
|
Adults and children (age 3-17) with X-linked hypophosphatemia will be treated with calcitriol therapy without phosphate supplementation.
Doses of calcitriol will be escalated and optimized in the first three months of the study.
Calcitriol is an oral medication taken once a day.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in serum phosphate in adults and children with XLH
Time Frame: up to 12 months
|
up to 12 months
|
|
Change from baseline in TmP/GFR in adults and children with XLH
Time Frame: up to 12 months
|
a measure of kidney resorption of phosphate
|
up to 12 months
|
Rickets score for children with XLH
Time Frame: up to 12 months
|
a score of rickets severity determined by reading x-rays of wrists and knees (10 point Thacher score with 0 being normal and 10 being severe)
|
up to 12 months
|
Change from baseline in nephrocalcinosis grade
Time Frame: up to 12 months
|
determine if there is change in amount of calcifications in the kidneys: graded from grade 0 (normal) to grade IV (stone formation, solitary focus of echos at the tip of the renal pyramid)
|
up to 12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Growth in children with XLH
Time Frame: up to 12 months
|
Z-score of growth
|
up to 12 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Eva Liu, MD, Massachusetts General Hospital and Brigham and Women's Hospital
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
March 28, 2019
Primary Completion (Estimated)
March 1, 2025
Study Completion (Estimated)
March 1, 2025
Study Registration Dates
First Submitted
November 19, 2018
First Submitted That Met QC Criteria
November 20, 2018
First Posted (Actual)
November 21, 2018
Study Record Updates
Last Update Posted (Actual)
March 12, 2024
Last Update Submitted That Met QC Criteria
March 11, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Kidney Diseases
- Urologic Diseases
- Nutrition Disorders
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Avitaminosis
- Deficiency Diseases
- Malnutrition
- Bone Diseases
- Metabolism, Inborn Errors
- Bone Diseases, Metabolic
- Renal Tubular Transport, Inborn Errors
- Calcium Metabolism Disorders
- Metal Metabolism, Inborn Errors
- Phosphorus Metabolism Disorders
- Vitamin D Deficiency
- Hypophosphatemia, Familial
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Rickets
- Familial Hypophosphatemic Rickets
- Hypophosphatemia
- Rickets, Hypophosphatemic
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Micronutrients
- Membrane Transport Modulators
- Vitamins
- Bone Density Conservation Agents
- Calcium-Regulating Hormones and Agents
- Vasoconstrictor Agents
- Calcium Channel Agonists
- Calcitriol
- Dihydroxycholecalciferols
Other Study ID Numbers
- 2016P001000
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on X-linked Hypophosphatemia
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Kyowa Kirin Co., Ltd.Active, not recruiting
-
Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaKorea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
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Kyowa Kirin, Inc.CompletedX-Linked HypophosphatemiaUnited States
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Kyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia (XLH)China
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