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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 56 (estimated)
• Sponsor: Rezolute · Academic / Other
• Sex: All
• Age: 3 Months – 45 Years
• Healthy volunteers: Not accepted

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 alone over 24 weeks and to evaluate the longer-term safety and efficacy of RZ358 during a subsequent open-label extension (OLE) period.

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 population, and as high as 1 in 2,500 in certain populations with substantial consanguinity. Despite improved recognition, there is no satisfactory treatment or cure for congenital HI. Current medical therapies for congenital HI are directed at reducing or eliminating insulin production and/or secretion from the beta-cell. These current medications, however, achieve suboptimal glycemic control and/or have undesirable side effects. A therapy which safely and effectively attenuates the activity of insulin would address an important unmet need for these and other conditions associated with HI. This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled (SOC alone), parallel-arm, efficacy, and safety study of RZ358 in participants with congenital HI who have not achieved adequate hypoglycemia control with reasonable attempts at using usual SOC medical therapy. The study will randomize approximately 48 participants (≥1 year to ≤45 years of age) in a 1:1 ratio into 2 dosing arms (5 or 10 mg/kg with) and further randomize participants within each dosing level in a 2:1 ratio to receive RZ358 as add-on to SOC or placebo as add-on to SOC. An additional open-label (OL) arm will be conducted in parallel for participants who are ≥3 months to \<1 year old (n=8), Upon completion of the pivotal treatment period (24-weeks), participants may roll-over to the OLE period at the discretion of the investigator and Sponsor.

Conditions

• Congenital Hyperinsulinism

Interventions

Type	Name	Description
DRUG	RZ358 (5 mg/kg) + SOC (Standard-of-Care) or Placebo + SOC	Participants ≥1 year old who receive SOC therapy and 5 mg/kg of RZ358 or placebo
DRUG	RZ358 (10 mg/kg) or Placebo + SOC	Participants ≥1 year old who receive SOC therapy and 10 mg/kg of RZ358 or placebo
DRUG	RZ358 (5-10 mg/kg) + SOC	Infant participants from ≥3 months to \<1 year old who receive SOC therapy + RZ358 starting at 5 mg/kg and increasing to 10 mg/kg of RZ358, as needed, per the protocol schedule

Timeline

• Start date: 2024-01-11
• Primary completion: 2025-11-14
• Completion: 2027-11-14
• First posted: 2024-01-17
• Last updated: 2026-01-08

Locations

18 sites across 13 countries: United States, Bulgaria, Denmark, France, Georgia, Germany, Greece, Oman, Qatar, Spain, Turkey (Türkiye), United Kingdom, Vietnam

Regulatory

• FDA-regulated drug study