Home › Trials › NCT06208215

RZ358 treatment for congenital hyperinsulinism

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients with Congenital Hyperinsulinism

Phase 3 Interventional Rezolute · NCT06208215

This study is testing if a new treatment called RZ358 can help people with congenital hyperinsulinism better control their blood sugar when added to their current medications.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment56 (estimated)
Ages3 Months to 45 Years
SexAll
SponsorRezolute Academic / other
Locations24 sites (Philadelphia, Pennsylvania and 23 other locations)
Trial IDNCT06208215 on ClinicalTrials.gov

What this trial studies

This Phase 3 pivotal study evaluates the efficacy and safety of RZ358 as an add-on therapy to standard-of-care for congenital hyperinsulinism. The trial involves a multicenter, randomized, double-blind, placebo-controlled design, comparing RZ358 to placebo over 24 weeks. Participants aged 3 months to 45 years who have not achieved adequate glycemic control with existing therapies will be enrolled. The study also includes an open-label extension for younger participants.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 3 months to 45 years with a clinical diagnosis of congenital hyperinsulinism who have not responded adequately to standard therapies.

Not a fit: Patients with liver function abnormalities or those who have not experienced sufficient hypoglycemia events may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve glycemic control for patients suffering from congenital hyperinsulinism.

How similar studies have performed: Other studies have explored treatments for congenital hyperinsulinism, but this specific approach with RZ358 is novel and has not been previously tested.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Philadelphia, Pennsylvania and 23 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Congenital HyperinsulinismHypoglycemiaGlucose Metabolism DisordersMetabolic DiseasesPancreatic DiseasesDigestive System DiseasesHyperinsulinism
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.