RZ358 Treatment for Congenital Hyperinsulinism

Launched by REZOLUTE · Jan 11, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called RZ358 for children and young adults with congenital hyperinsulinism (HI), a condition where the body produces too much insulin, leading to low blood sugar levels. The trial aims to see if adding RZ358 to standard treatments can improve blood sugar control over 24 weeks compared to standard treatments alone. Additionally, researchers will look at the long-term safety and effectiveness of RZ358 in a follow-up phase.

To be eligible for this trial, participants must be between 3 months and 45 years old and have a diagnosis of congenital HI. They should have tried standard treatments but still experience frequent low blood sugar events—at least three times a week. Participants will undergo regular monitoring to track their blood sugar levels and may receive RZ358 along with their usual care. It's important to note that certain health conditions and allergies may exclude individuals from participating. Overall, this trial offers a potential new option for managing a challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • At screening, aged ≥ 3 months and ≤ 45 years old.
• • An established clinical diagnosis of congenital HI (hyperinsulinism), with or without identification of a known monogenic variant by genetic testing.
• • Participant has failed to achieve adequate glycemic control with appropriate and reasonable trials of locally accepted and available Standard of Care (SOC) medical therapies (e.g., diazoxide and somatostatin analogs (SSAs)) per the judgment of the investigator.
• • Experiencing ≥ 3 hypoglycemia events per week by screening Self-Monitoring Blood Glucose (SMBG) and average daily percent time with hypoglycemia of ≥ 8% of the monitored screening Continuous Glucose Monitor (CGM) time.
• Exclusion Criteria:
• • Alanine aminotransaminase (ALT), aspartate aminotransaminase (AST), total bilirubin (TB), alkaline phosphatase (ALP), and gamma-glutamyl transferase (GGT) ≥ 1.5 × the upper limit of normal for the age-specific reference range, regardless of assessed significance.
• • Body mass index (BMI) ≥ 35 kg/m2 for participants aged 18 years and above, or BMI ≥ 99% (percentile) per Centers for Disease Control and Prevention growth charts for participants \> 12 and \< 18 years of age (no BMI exclusion for participants ≤ 12 years of age).
• • A known clinical diagnosis of diabetes or pre-diabetes, or a history of insulin dependency within 3 months of screening.
• • Average daily percent time with hyperglycemia ≥ 5% of the monitored screening continuous glucose monitoring (CGM) time.
• • Known allergy or sensitivity to RZ358 or any component of the drug.