Rifampin to Reduce Elevated Levels of Blood and Urine Calcium in Patients With Inactivating Mutations in the CYP24A1 Gene
Who is this study for? Patients with hypercalcemia and/or hypercalciuria in with biallelic inactivating mutations of the CYP24A1 gene
What treatments are being studied? Rifampin
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 65
Healthy Volunteers: f
View:
• 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.
Locations
United States
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Michael A Levine, MD
levinem@chop.edu
267-426-3907
Backup
Vashisht Arshanapally
arshanapav@chop.edu
267-426-7482
Time Frame
Start Date: 2018-07-25
Estimated Completion Date: 2030-12
Participants
Target number of participants: 60
Treatments
Experimental: All Subjects
SingleArm: Escalating doses of rifampin (5 and 10 mg/kg/day) (SingleArm)
Authors
Related Therapeutic Areas
Sponsors
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Leads: Children's Hospital of Philadelphia