Hypoglycemia associated with congenital hyperinsulinism MedDRA version: 20.1 Level: LLT Classification code 10077227 Term: Hyperinsulinemic hypoglycemia System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 20.0 Level: LLT Classification code 10020644 Term: Hyperinsulinism NOS System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 20.0 Level: PT Classification code 10061211 Term: Hyperinsulinism System Organ Class: 10027433 - Metabolism a
Conditions
Interventions
Product Code: RZ358
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not Available
Current Sponsor code: RZ358
Other descriptive name: fully human IgG2 monoclonal antib
Sponsors
Rezolute, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Provide written informed consent and, as applicable, assent, before any study specific procedures are performed 2. At Screening, aged =2 years and =45 years 3. An established clinical diagnosis, with or without a genetic diagnosis, of congenital hyperinsulinism 4. Patient is currently receiving SOC medications (i.e corticosteroids, diazoxide, lanreotide, sirolimus, and octreotide) and/or nutritional supplementation for CHI, with at least 2 months of stable treatment; OR is not on treatment, at the time of Screening. 5. Glucose values =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any out-of-range laboratory value at Screening that has not been reviewed, approved, and documented as not clinically significant by the Investigator, with the exception of liver function tests for total bilirubin, ALT, AST, and ALP, which must be within 1.5X the upper limit of normal (ULN) for the reference range 2. Body mass index = 35 kg/m2 for patients aged 18 years and above and, for patients aged less than 18 years, BMI = 95% percentile for age 3. History of malignancy within 3 years before Screening other than carcinoma in situ of the cervix or adequately treated non-metastatic squamous or basal cell carcinoma of the skin 4. History of seropositivity for HIV antibody, hepatitis B, or hepatitis C antibody 5. Major general surgery within 3 months before Screening or anticipated during the study period 6. Use of systemic corticosteroids within 30 days before Screening 7. Known allergy or sensitivity to RZ358 or any component of the study drug 8. Treatment with an investigational drug or device within 30 days or 5 half-lives of the investigational drug before Day 1 of Week 1, whichever is longer. Participation in registries and purely diagnostic studies is allowed. 9. Female patients who are pregnant, planning to become pregnant during the course of the study, have recently delivered (within 3 months before Screening), or are breastfeeding 10. Male patients who are planning a pregnancy with a female partner during the course of the study or within 105 days after administration of study drug 11. Any organ condition, concomitant disease (eg, psychiatric illness, severe alcoholism, or drug abuse, cardiac, hepatic, or kidney disease), or other abnormality that itself, or the treatment of which, could interfere with the conduct of the study (eg, may affect absorption, distribution, metabolism, or elimination of the study drug) or that, in the opinion of the Investigator and/or Sponsor’s medical monitor, would pose an unacceptable risk to the patient in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objectives of this study are to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and glycemic efficacy of multiple ascending doses of RZ358 administered for 8 weeks in patients with hyperinsulinemic hypoglycemia due to congenital hyperinsulinism (CHI).;Secondary Objective: not applicable;Timepoint(s) of evaluation of this end point: Measured at various timepoints (see schedule of events);Primary end point(s): • Glycemic efficacy of RZ358 as evaluated by the average daily percent time within a glucose target range of 70-180 mg/dL (3.9-10 mmol/L) by continuous glucose monitoring (CGM); • Repeat-dose safety and tolerability of RZ358 administered for a total of 8 weeks; • Repeat-dose pharmacokinetics of RZ358. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Average weekly incidence of hypoglycemia (< 70 mg/dL) by self-monitored blood glucose (SMBG) • Average daily duration and percent time with hypoglycemia at thresholds of: < 70 mg/dL (3.9 mmol/L) < 60 mg/dL (3.3 mmol/L) < 50 mg/dL (2.8 mmol/L) by CGM • Average daily hypoglycemia incidence (event rate) at each of the specified glucose thresholds by CGM; • Average 8h overnight percent time in glucose target range of 70-180 mg/dL (3.9-10 mmol/L) by CGM; • Occurrence of hypoglycemia during a 12h overnight fasting challenge ;Timepoint(s) of evaluation of this end point: Measured at various timepoints (see schedule of events) | — |
Countries
Bulgaria, Croatia, Denmark, Germany, Israel, Serbia, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trials Info
Rezolute, Inc
Outcome results
None listed