CompletedPhase 2

An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism

United States, Bulgaria, Canada23 participantsStarted 2020-02-24

Plain-language summary

The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).

Who can participate

Age range

2 Years – 45 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female age 2-45 years old (except age 12-45 in US) with an established clinical diagnosis of congenital hyperinsulinism * Able to provide written informed consent or, as applicable, assent * Confirmed hypoglycemia as assessed by CGM, SMBG, and clinical evaluation, during Screening * Willingness to use contraception if of child-bearing potential Exclusion Criteria: * Out of range blood work for study entry * Body Mass index outside of study entry criteria * History of malignancy * Clinically significant diseases, seropositivity for HIV, hepatitis B or C antibody * Use of systemic corticosteroids within 30 days before Screening * Known or suspected allergy to the study drug * Recent use of an investigational drug or treatment, or participation in an investigational study * Pregnant or lactating women * History of drug abuse or excessive alcohol use

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT)

Timeframe: 8 weeks

Median Percent Change of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM From Baseline (BL)

Timeframe: 8 weeks

Repeat Dose Pharmacokinetics of RZ358

Timeframe: Pre dose Weeks 1,3,5,7, 1-hr post dose Week 1 and Week 7, and Follow up on Days 14, Day 28, Day 42, and Day 105

Trial details

NCT IDNCT04538989

SponsorRezolute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-04-05

Results submitted2024-11-25

View on ClinicalTrials.gov More Congenital Hyperinsulinism trials

Who can participate

Age range

2 Years – 45 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT)

Timeframe: 8 weeks

Median Percent Change of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM From Baseline (BL)

Timeframe: 8 weeks

Repeat Dose Pharmacokinetics of RZ358

Timeframe: Pre dose Weeks 1,3,5,7, 1-hr post dose Week 1 and Week 7, and Follow up on Days 14, Day 28, Day 42, and Day 105