Status:
ACTIVE_NOT_RECRUITING
RZ358 Treatment for Congenital Hyperinsulinism
Lead Sponsor:
Rezolute
Conditions:
Congenital Hyperinsulinism
Eligibility:
All Genders
3-45 years
Phase:
PHASE3
Brief Summary
The Phase 3 pivotal study is designed to evaluate the efficacy and safety of RZ358 for the treatment of congenital hyperinsulinism (HI) as add-on to standard-of-care (SOC) therapy compared to SOC alon...
Detailed Description
Congenital hyperinsulinism (HI) is the most common cause of recurrent hypoglycemia in neonates and infants with an incidence of approximately 1 in 25,000 to 1 in 50,000 live births in the general popu...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 11 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 14 2027
Estimated Enrollment :
56 Patients enrolled
Trial Details
Trial ID
NCT06208215
Start Date
January 11 2024
End Date
November 14 2027
Last Update
January 8 2026
Active Locations (18)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
2
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
3
Rezolute Investigative Site, Varna, Bulgaria
Varna, Bulgaria, 9010
4
Rezolute Investigative Site, Odense, Denmark
Odense, Denmark, 5000