What this study is
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.
What's being tested
- Rifampin
Intervention · DRUG
Rifampin 5 mg/kg (max 300 mg) daily for 8 weeks, followed by rifampin 10 mg/kg (max 600 mg) daily for 8 weeks.
Study details
- Official title
- Rifampin to Reduce Elevated Levels of Blood and Urine Calcium in Patients With Inactivating Mutations in the CYP24A1 Gene
- Sponsor
- Children's Hospital of Philadelphia
- Conditions
- Idiopathic Infantile Hypercalcaemia - Severe Form · Genetic Disease · Hypercalcemia, Idiopathic, of Infancy · Hypercalciuric Hypercalcemia · Idiopathic Infantile Hypercalcemia - Mild Form · Hypercalciuria
- Enrolling
- 60 people
- Starts
- 2018-07-25
- Est. completion
- 2030-12-01
- Design
- Open-label, single group, Phase 2
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 6 Months – 70 YearsSex ALLHealthy volunteers Not acceptedYou may qualify if
- Males or females age 6 months to 70 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.
You can't join if
- 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
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (1)
1 openChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
- Michael A Levine, MD · Contact267-426-3907levinem@chop.edu
- David Weber, MD · Contact203-843-0021PINNEYS@chop.edu
- David Weber, MD · Sub-investigator
- Michael A Levine, MD · Principal investigator
Recruiting
Study team
Sponsor
Children's Hospital of Philadelphia
Study ID 16-013429
Principal investigator
Michael A Levine, MD
Children'sHospital of Philadelphia
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.