Calcitriol Monotherapy for X-Linked Hypophosphatemia (NCT03748966) | Clinical Trial Compass
CompletedEarly Phase 1
Calcitriol Monotherapy for X-Linked Hypophosphatemia
United States16 participantsStarted 2019-03-28
Plain-language summary
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.
Who can participate
Age range
3 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Serum Phosphate in Adults and Children With XLH Between Baseline and 12 Months
Timeframe: baseline and 12 months
2
Change From Baseline in TmP/GFR in Adults and Children With XLH