NCT03384121

The Antibiotic Rifampin to Reduce High Levels of Blood and Urine Calcium in IIH

Status unknown Phase 1 Last updated 31 August 2021

What this trial tests

Phase 1 trial testing Rifampin 150 mg, 300 mg capsules and 25 mg/mL oral suspension in Idiopathic Infantile Hypercalcemia - Mild Form in 5 participants. Status unknown.

Timeline

22 February 2018

Primary endpoint
31 December 2021

31 December 2021

Quick facts

Lead sponsor	The Hospital for Sick Children
Phase	Phase 1
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	22 February 2018
Primary completion	31 December 2021
Estimated completion	31 December 2021
Sites	1 location across Canada

Drugs / interventions tested

• Rifampin 150 mg, 300 mg capsules and 25 mg/mL oral suspension — full drug profile →

Conditions studied

• Idiopathic Infantile Hypercalcemia - Mild Form — all drugs for Idiopathic Infantile Hypercalcemia - Mild Form →

Sponsor

The Hospital for Sick Children

Who can join

Adults 6 Months to 17, any sex, with Idiopathic Infantile Hypercalcemia - Mild Form. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Idiopathic infantile hypercalcemia(IIH) is a rare,genetic disorder of mineral metabolism. Biallelic loss of functions mutations of CYP24A1, the gene encoding the 24-hydroxylase enzyme that represents the principal pathway for inactivation of vitamin D metabolites, cause the most common and severe form of IIH.Investigators have preliminary data supporting a novel therapeutic approach to suggest rifampin as an investigational drug to induce over-expression of CYP3A4, an important enzyme that provides an alternate catabolic pathway for inactivation of vitamin D metabolites. In this study, investigators will recruit 5 patients with biallelic inactivating mutations of CYP24A1. Participants will be followed prospectively for a total 6-11 months. This will include 2 months of observation, 2 months of receiving the starting dose of rifampin, followed by 2 month washout phase. Efficacy of the starting dose of rifampin will be determined prior to proceeding only in non responders to the escalation dose of rifampin 10mg/kg/day.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.