NCT03301038 Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
| NCT ID | NCT03301038 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Idiopathic Infantile Hypercalcaemia - Severe Form |
| Study Type | INTERVENTIONAL |
| Enrollment | 60 participants |
| Start Date | 2018-07-25 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 60 participants in total. It began in 2018-07-25 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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: * 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
Contact & Investigator
Michael A Levine, MD
PRINCIPAL INVESTIGATOR
Children'sHospital of Philadelphia
Frequently Asked Questions
Who can join the NCT03301038 clinical trial?
This trial is open to participants of all sexes, aged 6 Months or older, up to 65 Years, studying Idiopathic Infantile Hypercalcaemia - Severe Form. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03301038 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT03301038 currently recruiting?
Yes, NCT03301038 is actively recruiting participants. Contact the research team at levinem@chop.edu for enrollment information.
Where is the NCT03301038 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT03301038 clinical trial?
NCT03301038 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Michael A Levine, MD at Children'sHospital of Philadelphia. The trial plans to enroll 60 participants.