Sirolimus for the Treatment of Hyperinsulinism
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|ClinicalTrials.gov Identifier: NCT02524639|
Recruitment Status : Withdrawn (Due to strict inclusion and exclusion criteria no subjects were enrolled)
First Posted : August 17, 2015
Last Update Posted : June 13, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hyperinsulinism||Drug: Sirolimus||Phase 1 Phase 2|
Treatment options for children with diffuse adenosine triphosphate-sensitive potassium (KATP) channel hyperinsulinism (KATPHI) are limited and most of them require a near-total pancreatectomy to control the hypoglycemia. However, at least 40% of these children continue to have persistent hypoglycemia after surgery and their long-term outcomes are complicated by the development of diabetes.
There is evidence that suggests that mammalian target of rapamycin (mTOR) inhibitors are useful in controlling the hypoglycemia in hyperinsulinemic hypoglycemia. But before adapting this as standard therapy for children with hyperinsulinism, a carefully controlled study of the efficacy and safety of sirolimus for hyperinsulinism is clearly needed.
Sirolimus is an mTOR inhibitor, which is FDA-approved for the prophylaxis of organ rejection in patients age 13 years and older receiving kidney transplantation. This is an open label pilot study to assess the effect, safety and tolerability of sirolimus in infants with diazoxide-unresponsive HI due to mutations in the genes encoding the KATP channels. Subjects will be treated with sirolimus for 6 weeks.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of the Efficacy and Safety of Sirolimus in the Treatment of Congenital Hyperinsulinism.|
|Estimated Study Start Date :||August 12, 2015|
|Actual Primary Completion Date :||May 29, 2018|
|Actual Study Completion Date :||May 29, 2018|
All enrolled subjects will receive Sirolimus 1 mg/m2/day twice a day for 6 weeks.
Subjects will receive 1 mg/m2/day orally for 6 weeks. Maintenance dose will be titrated up or down by 0.25-0.5 mg/m2/day every 4 days. Serum concentration will be checked on day 4 after initial therapy and 4 days after any dose adjustment. Levels will be checked at lease once a week during the duration of the study. Target serum concentration range is 5-10 ng/mL.
Other Name: Rapamune
- Number of children off intravenous dextrose support [ Time Frame: 6 weeks ]
- Change in number hypoglycemic episodes per child per day [ Time Frame: 6 weeks ]
- Plasma insulin levels during fasting [ Time Frame: 8 hours ]
- Number of participants with Adverse Events [ Time Frame: 6 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02524639
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Diva De Leon, MD||Children's Hospital of Philadelphia|