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Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria

NCT03301038 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2025-07-28

No results posted yet for this study

Summary

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.

Conditions

  • Idiopathic Infantile Hypercalcaemia - Severe Form
  • Genetic Disease
  • Hypercalcemia, Idiopathic, of Infancy
  • Hypercalciuric Hypercalcemia
  • Idiopathic Infantile Hypercalcemia - Mild Form
  • Hypercalciuria

Interventions

DRUG

Rifampin

Rifampin 5 mg/kg (max 300 mg) daily for 8 weeks, followed by rifampin 10 mg/kg (max 600 mg) daily for 8 weeks.

Sponsors & Collaborators

Principal Investigators

  • Michael A Levine, MD · Children'sHospital of Philadelphia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-07-25
Primary Completion
2026-12-31
Completion
2030-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03301038 on ClinicalTrials.gov