sunRIZE Study: RZ358 Clinical Trial for Hyperinsulinism
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Description
This clinical trial is for patients with congenital hyperinsulinism (HI) who are experiencing low blood sugar on their current treatment. The purpose of this study is to find out if the study drug, named RZ358, helps improve blood sugar levels compared to current HI treatments. RZ358 is an experimental drug which the FDA has approved for use in this clinical trial, but it has not approved the study drug to be given outside of clinical research testing. The study drug is a specialized protein (called a monoclonal antibody) meant to decrease the effect insulin has on lowering blood sugar.
Participation will last for up to 33 weeks or up to 3 years overall for subjects who would like to join the open-label extension phase. This includes a 5-week Screening Period, a Treatment Period that lasts 24 weeks, and then either an 8-week Follow-up Period or a 2-year Extension Period where participants can continue to receive the study drug. Individuals do not need to participate in the 2-year extension part of the study to take part in the treatment and monitoring period. During the treatment period, participants will be given the study drug or a placebo. A placebo is an inactive substance used to compare how well the study drug works. Only study drug and not placebo is given during the extension period.
Participants in this trial will:
- Receive the study drug or a placebo 7 times during the treatment period
- Receive the study drug for up to 2 years in the optional extension period
- Have electrocardiograms (ECGs)
- Have ultrasounds of their liver
- Wear a continuous glucose monitor (CGM) and take fingerstick glucose checks
- Have research blood tests
- Complete study diaries and surveys
The main risks of this study are from the study drug. These include infusion reactions (redness, tenderness, irritation at the infusion site), headaches, dizziness, hypoglycemia (low blood sugar), upper respiratory infections (colds), and excess body hair growth. Participants may benefit if the study drug proves to be more effective than current HI medications.
Eligibility and criteria
What to expect
This study has 4 parts: a Screening Period (about 5 weeks), a Treatment Period (about 6 months), an Extension Period (about 8 weeks), and a Follow-up Period (about 2 years). In total, there are approximately 64 outpatient study visits over approximately 3 years. This study uses a double-blind, placebo-controlled design. This means that participants will be randomly assigned ("by chance" or "like the flip of a coin") to receive either study drug infusion or the placebo infusion during the Treatment Period; neither the participant nor the study doctor will know which infusion is given in this period. After the Treatment Period, participants may then enter an Extension Period to receive unblinded study drug for up to approximately 2 years.
- Screening Period: The screening visit and 5-week screening period helps us determine if participants are eligible for the study. A screening visit includes reviewing medical history, taking blood and urine samples, doing a physical exam (with vitals), and completing an ECG and liver ultrasound.
- Treatment Period: This period involves participants receiving study drug or placebo every other week 3 times and then study drug or placebo every month for 4 more times. This is a total of 7 infusions over the course of about 6 months.
- Follow-up Period: After completing the Treatment Period, participants can choose to stop receiving the study drug or placebo and enter the Follow-up Period. During this 8-week period, participants come back to CHOP one time to complete study procedures including bloodwork, ECG, liver ultrasound, and questionnaires.
- Extension Period: Participants may also enroll in the Extension Period to receive study drug for up to approximately 2 years. During this Period, the study doctor may increase or decrease the study drug dosage and possibly decrease other HI medications.
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