Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
Recruiting
Phase 2
Interventional Study
Idiopathic Infantile Hypercalcaemia - Severe Form
Genetic Disease
Hypercalcemia, Idiopathic, of Infancy
Hypercalciuric Hypercalcemia
Idiopathic Infantile Hypercalcemia - Mild Form
Hypercalciuria
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 Months – 65
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- Children's Hospital of Philadelphia · Other
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About This Trial
This study evaluates the efficacy of rifampin in the treatment of hypercalcemia and/or hypercalciuria in participants with at least one inactivating mutation of the CYP24A1 gene. Eligible subjects will receive rifampin for a total of 16 weeks during this study.
Trial Locations
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Eligibility Criteria
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…
Contacts