RZ358 for Congenital Hyperinsulinism

(sunRIZE Trial)

Recruiting at 22 trial locations

Overseen By Gopal Saha, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Rezolute

Must be taking: Standard of care

Disqualifiers: Diabetes, Pre-diabetes, High BMI, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

56 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications. It mentions that RZ358 is added to standard-of-care treatments, so you may continue your existing medications.

What is the purpose of this trial?

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.

Research Team

Gopal Saha, MD

Principal Investigator

Rezolute

Eligibility Criteria

This trial is for individuals with congenital hyperinsulinism, a condition causing excessive insulin production. Participants should be currently receiving standard treatments without sufficient control of their condition.

Inclusion Criteria

I am between 3 months and 45 years old.

I have been diagnosed with congenital hyperinsulinism, with or without a known genetic cause.

I have low blood sugar levels 3 or more times a week.

Exclusion Criteria

I am allergic to RZ358 or its ingredients.

My BMI is 35 or higher, or I am a teen with a BMI in the top 1% for my age.

My blood sugar is often high, more than 5% of the time.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive RZ358 or placebo as add-on to standard-of-care therapy for congenital hyperinsulinism

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of RZ358 treatment long-term

Up to two years

Treatment Details

Interventions

• RZ358 Treatment

Trial Overview The study tests RZ358 at different doses (5 mg/kg and 10 mg/kg) added to the usual care versus a placebo plus usual care over six months. It also looks at how safe and effective RZ358 is over a longer period.

Participant Groups

Group I: Open Label Arm, SoC + RZ358 (start 5mg/kg and increase to 10 mg/kg per protocol schedule Experimental Treatment 1 Intervention

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

Group II: SoC (Standard-of-Care) + RZ358 (5 mg/kg) or Placebo Placebo Group 1 Intervention

Participants ≥1 year old who receive SOC therapy and 5 mg/kg of RZ358 or placebo

Group III: SoC + RZ358 (10 mg/kg) or Placebo Placebo Group 1 Intervention

Participants ≥1 year old who receive SOC therapy and 10 mg/kg of RZ358 or placebo

Who Is Running the Clinical Trial?

Rezolute

Lead Sponsor

Trials

4

Recruited

230+