Rezolute

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This trial is not actively recruiting.

This trial has completed recruitment on this platform, and is no longer accepting new referrals.

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

This study is evaluating the safety, tolerability, and glucose-raising effects of RZ358 in patients with congenital hyperinsulinism (HI).

Trial ID: NCT04538989

Rezolute is committed to ensuring the safety of all study participants and has taken steps to reduce the burden that comes with study participation as much as possible. The study is open to anyone that meets the entry criteria and is willing to travel to a clinical trial site location that is participating in the RIZE study. The RIZE study provides participants and a family member with travel, lodging and food support during the study and follow up visits.

Trial Details

We want to learn if RZ358 can be used to prevent severe, persistent low blood sugar in patients with congenital hyperinsulinism (HI). There is a significant need to develop safer, better tolerated and more effective therapies aimed at preventing chronic and recurrent hypoglycemia (low blood sugar) in HI.

Congenital HI is a rare genetic condition, which most commonly presents at birth, and causes excessive and unregulated insulin release from the pancreas. This can leave patients vulnerable to frequent, potentially dangerous episodes of low blood sugar. In the developing child, the brain is particularly vulnerable to these low blood sugar episodes, requiring intensive monitoring and intervention to minimize potential neurologic damage. There is no cure for HI and current therapies are either ineffective or are not well tolerated from a safety perspective. If removal of the pancreas is required, this may be associated with short and long-term complications, and often results in insulin-dependent diabetes.

RZ358 is an intravenously administered monoclonal antibody that was developed and tailored for the potential treatment HI.
RZ358 has a unique mechanism of action which dampens the effects of elevated insulin, thus having the potential to treat low blood glucose levels in patients with congenital HI.

This clinical trial is designed to evaluate the safety and effectiveness of RZ358 in patients with congenital HI whose hypoglycemia is inadequately controlled on existing therapies. To determine if you can take part in the study, you will need to visit a study center to undergo medical tests and assessments. The eligibility visit and screening period is up to 5 weeks before the start of the study.

Estimated Enrollment

24-32 Participants

Phase

Eligibility Criteria

Participants must:

Be male or female age 12-45 years old in the US or age 2-45 years old outside the US, with an established clinical diagnosis of HI
Experience frequent hypoglycemia (multiple times a week) as assessed by continuous glucose monitoring, home glucometer, and clinical evaluation during screening
Be able to provide written informed consent or, as applicable, assent
Be willing to use contraception if of child-bearing age

Participants must not:

Out-of-range blood work for certain labs for study entry
A history of cancer
Clinically significant diseases, HIV or Hepatitis
Used an oral or injected steroid within 30 days before screening
Known or suspected allergy to the study drug (RZ358)
Recently used an investigational drug or treatment, or participated in an investigational study
Current pregnancy or be breastfeeding
A history of drug abuse or excessive alcohol use

What Will Happen in This Study

Once a participant is enrolled, RZ358 will be administered via a 30-minute infusion weekly or bi-weekly for a total treatment period of 8 weeks and a post-treatment follow-up period of 13 weeks.

By taking part in the study, you will receive the following at no charge:

A comprehensive evaluation of your overall health
RZ358 study drug
Study-related medical care such as physical examinations, hepatic ultrasound (pictures of your liver) and blood and urine tests

If you choose to participate and you or your child is eligible, you will be directed to one of the study centers taking part in this clinical trial. Travel assistance and lodging is provided for patients/families that require support to and from the specific centers during study participation.

You will need to travel to one of the study clinics taking part in this clinical trial.

Sites are located in the cities/states identified with the red pin:

Frequently Asked Questions (FAQs)

Will I get paid to be a part of this clinical trial?

All study-related services, including travel/lodging are provided at no cost. You also may be paid for your time. The individual study site will review all these details with you. Also, you do not have to pay to be in the study.

Does this study use a placebo?

No. This study is NOT using a placebo. All study participants will remain on their current HI standard of care. In the RIZE study the safety and efficacy of RZ358 will be evaluated during the study as add-on therapy to other therapies that the patient may already be taking to treat their hypoglycemia due to HI

How can I check to see if I am eligible for this trial?

You can check if you meet the basic requirements for this trial by completing a set of screening questions. You can find these on this web page by clicking the ‘Get Started’ button. Full eligibility will be determined by the RIZE study site. Please note that you are not obligated to enroll in the study. You can exit the RIZE prescreening website at any time if you wish.

What does a typical research study look like?

Clinical trials help determine the safety and effectiveness of new treatments. A research plan or protocol is created that outlines how trial participants may receive investigational medications, devices, or procedures.

Clinical trials are performed according to strict government and ethical guidelines. These guidelines help to ensure patient rights are protected while information about the new treatment is collected.

Can I quit the trial at any time?

Study volunteers have the right to drop out of the study at any time for any reason, or no reason at all. Your study doctor, Sponsor or Regulatory Authority also has the right to discontinue a study volunteer at any time for the participant’s safety, or if the participant no longer meets the study requirements.