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Can a vitamin d pill improve bone health in rare genetic disease?

NCT ID NCT03748966

Disease control Sponsor: Massachusetts General Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested whether giving only calcitriol (a form of vitamin D) to 16 children and adults with X-linked hypophosphatemia (XLH) could raise blood phosphate levels and improve bone growth without causing kidney stones. Participants took increasing doses of calcitriol for one year. The goal was to see if this simpler treatment could help manage the disease and support growth in children.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

16 people

The number who actually took part.

Started

Mar 2019

Finished

Feb 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

bone disorder hypophosphatemic rickets rickets X-linked dominant hypophosphatemic rickets X-linked hypophosphatemic rickets

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hypophosphatemic rickets Hypophosphatemic rickets, X-linked dominant X-linked hypophosphatemia

Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.