A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and clinical pharmacology of a single dose pharmacokinetics and pharmacodynamics of XOMA 358 in subjects with hypoglycemia associated with congenital hyperinsulinism.

Official Title
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A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism (HI)

Conditions
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Congenital Hyperinsulinism

Intervention / Treatment
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* Drug: Cohort 1
* Drug: Cohort 2
* Drug: Cohort 3
* Drug: Cohort 4


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical diagnosis of congenital hyperinsulinism Duration of glucose levels less than 70mg/dL, by CGM, for an average of at least 120 minutes a day across baseline Days -3, -2, and -1 with no single duration less than 60 minutes on any of the baseline Days -3, -2, or -1, unless the subject received rescue treatment. Can safely washout of background medications used to treat hyperinsulinism. Exclusion Criteria: Use of any agent, such as diazoxide, octreotide, chronic systemic glucocorticoids, or β agonists that may affect glucose metabolism Body Mass Index ≥ 35 kg/m2 Pregnant females, females planning to become pregnant during the course of the study, females who have delivered 3 months before screening, or breastfeeding Male subjects who are planning a pregnancy with a female partner during the course of the study or within 4 months after administration of study drug.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Cohort<br>XOMA 358 dose level A, dose level B, dose level C, and dose level D. | Drug: Cohort 1<br>* XOMA 358 single dose level A administered by an intravenous infusion<br>Drug: Cohort 2<br>* XOMA 358 single dose level B administered by an intravenous infusion<br>Drug: Cohort 3<br>* XOMA 358 single dose level C administered by an intravenous infusion<br>Drug: Cohort 4<br>* XOMA 358 single dose level D administered by an intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events | Safety assessed by treatment-emergent adverse events | 42 days | 
| Change from baseline in glucose levels as measured using a continuous glucose monitor | Safety assessed by pre- and post-treatment measurements of vital signs and laboratory test results | 42 days | 
| Fasting and post prandial blood glucose levels | Assessment of blood glucose collected at time points specified in the protocol | 42 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hypoglycemia, Congenital Hyperinsulinism