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Common antibiotic could tame rare calcium disorder

NCT ID NCT03301038

Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests whether the antibiotic rifampin can safely lower high calcium levels in the blood and urine of people with a rare genetic mutation in the CYP24A1 gene. The condition can cause kidney stones and other problems. Sixty participants aged 6 months to 65 years will take rifampin for 16 weeks, with regular monitoring of calcium and related markers.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Rifampin (an antibiotic)
What this could lead to
If it works, this could offer a way to manage dangerously high calcium levels in people with this rare genetic condition, reducing kidney damage and other complications.
What could go wrong
This is a small, early-phase trial with only 60 people. Rifampin may not lower calcium enough, or side effects like liver issues or allergic reactions could limit its use.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2018

Expected to finish

Dec 2030

An estimate. End dates often move.

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

6 months to 65 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: * Males or females age 6 months to 65 years. * at least one mutations of CYP24A1 * Serum and/or urinary calcium above the normal reference range for age * Serum PTH concentration \<20 pg/ml * Elevated or normal serum concentration of 1,25-dihydroxyvitamin D3. Exclusion Criteria: * Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures. * Allergy to rifampin or related medications * Current therapies with medications that have significant drug-drug interactions with rifampin, defined as a medication considered to interact with CYP3A4 or CYP3A5 and either induce or inhibit expression or function of these P450 enzymes. By "drug-drug" interactions we are looking for medications that will affect metabolism or action of rifampin as exclusionary, not medications that will be affected by rifampin. * Pregnancy or breastfeeding * Laboratory abnormalities that indicate clinically significant hepatic, or renal disease: * Aspartate Aminotransferase (AST/SGOT) \> 2.0 times the upper limit of normal Alanine aminotransferase (ALT/SGPT) \> 2.0 times the upper limit of normal Total bilirubin \> 2.0 times the upper limit of normal Creatinine \> 2.0 times the upper limit of normal

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Conditions

The condition(s) this trial relates to.

hereditary disease hypercalcemia disease Hypercalcemia, Idiopathic, of Infancy hypercalcemia, infantile Hypercalciuria Hypercalciuric Hypercalcemia idiopathic hypercalciuria nephrocalcinosis

As listed by the trial registrant

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

Genetic disease Hypercalcemia, idiopathic, of infancy Hypercalciuria Hypercalciuric hypercalcemia Idiopathic infantile hypercalcaemia - severe form Idiopathic infantile hypercalcemia - mild form

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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