Phase 3 study to evaluate the efficacy and safety of RZ358 for Congenital Hyperinsulinism
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients with Congenital Hyperinsulinism
IRAS ID
1007936
Contact name
Katja Schuster
Contact email
Sponsor organisation
Rezolute, Inc
ISRCTN Number
NA
Clinicaltrials.gov Identifier
NA
Research summary
Current medical therapies for congenital hyperinsulinism (CHI) work to turn down or turn off insulin production and/or secretion. These medications are not enough to control blood sugars and/or have bad side effects. A therapy which safely and successfully controls how insulin acts in the body would meet an important unmet need in congenital hyperinsulinism. RZ358 was specifically designed to treat CHI in a new and different way than previous medicines. The goals of this study are to test how well and how safely RZ358 treats CHI. The study will measure the effect of RZ358 on low blood sugar events through finger stick blood sugars checks (BGM) and time below normal range using a continuous glucose monitor (CGM) as well as other measures of low blood sugar levels over 24 weeks of treatment. The study will also measure the levels of RZ358 and effects of the therapy on other important blood tests. Additionally, the sponsor will look at how RZ358 affects certain important clinical outcomes that are connected to having low blood sugar as well as the quality of life of patients. The study will consist of a screening period, a double-blind, placebo-controlled treatment period (or Open Label arm for infants) of RZ358, and either an Open Label Extension period OR an end of study follow-up period. Overall, the study will enroll about 56 participants. The study will assign in random order 48 participants (≥1 year to ≤45 years of age) into 2 dosing arms (5 or 10 mg/kg) and further assign in random order participants within each dosing level to receive RZ358 or placebo. Additionally, infant participants (≥3 months to <1 year old) will be enrolled into an Open label arm (meaning all infant participants in this group will receive RZ358. Thus, the study will comprise the following overall treatment groups.
REC name
London - Hampstead Research Ethics Committee
REC reference
23/LO/0786
Date of REC Opinion
9 Nov 2023
REC opinion
Further Information Favourable Opinion