Sirolimus for the Treatment of Hyperinsulinism

Withdrawn before enrolling · Phase 1/Phase 2

Conditions studied: Hyperinsulinism

In brief

The purpose of this pilot study is to generate data to assess feasibility of study design/procedures and for formal sample size estimation for a larger multicenter study of the efficacy and safety of sirolimus in infants with medically-unresponsive congenital hyperinsulinism (HI) due to inactivating mutations of adenosine triphosphate-sensitive potassium (KATP) channels.

Key facts

Study ID

NCT02524639

Run by

Children's Hospital of Philadelphia

People needed

0

Starts

2015-08-12

Expected to finish

2018-05-29

Last updated by the study team

2018-06-13

Who can join

Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males or females age ≥14 days to 12 months.
• Confirmed diagnosis of hyperinsulinism.
• Mutation analysis results demonstrating bi-allelic mutations in either ABCC8 or KCNJ11.
• Failure to respond to maximal dose of diazoxide (15 mg/kg/day), if diazoxide is indicated.
• Unable to wean intravenous dextrose after at least 3 days of diazoxide therapy and/or
• Persistent hypoglycemia after at least 3 days of diazoxide therapy
• High glucose infusion rate requirement (greater or equal to 10 mg/kg/min).
• Parental/guardian permission (informed consent).

You may not qualify if…

• Infants with suspected or confirmed focal hyperinsulinism who are candidates for surgical resection
• Current therapy with diazoxide. Subjects may be eligible for participation 48 hrs after discontinuation of diazoxide.
• Laboratory abnormalities that indicate clinically significant hematologic, hepatobiliary, or renal disease:
• AST/SGOT > 2.5 times the upper limit of normal
• ALT/SGPT > 2.5 times the upper limit of normal
• Total bilirubin > 2.5 times the upper limit of normal
• Hemoglobin < 9 gm/dL
• White blood cell count < 3,000/ mm3
• Platelet count < 100,000/mm3
• Creatinine > 2.5 times the upper limit of normal
• Evidence of active infection.
• Evidence of cardiac or respiratory failure.
• Known immune deficiency.
• Preterm (< 37 week gestation at birth).
• Treatment with immunosuppressants.
• Treatment with any drug known to interact significantly with sirolimus (strong inducers and strong inhibitors of CYP3A4 and P-gp with risk category D and X) including:
• Cyclosporine, clozapine, conivaptan, crizotinib, dabrafenib, dipyrone, boceprevir, echinacea, efavirenz, enzalutamide, fluconazole, fosphenytoin, fusidic acid, idelalisib, leflunomide, lomitapide, mifepristone, mitotane, natalizumab, nelfinavir, phenytoin, pimecrolimus, pimozide, posaconazole, roflumilast, St Johns Wort, stiripentol, tacrolimus, telaprevir, tofacitinib, rifampin, rifabutin, ketoconazole, voriconazole, itraconazole, erythromycin, telithromycin, clarithromycin
• Any investigational drug use within 5 half-lives of the drug prior to initiation of therapy.
• Subjects who had participated in other investigational drug studies will be eligible to participate after 5 half-lives from the last dose of the investigational agent and have recovered from acute investigational agent associated toxicity
• History of surgical procedure within 8 weeks of enrollment.
• Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.

Where it is running

• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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