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

May 9, 2025 updated by: Rezolute

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

There is a significant unmet medical need to develop new therapies aimed at preventing chronic recurrent hypoglycemia in congenital HI, the most common cause of persistent hypoglycemia in children. RZ358 is a human mAb that allosterically attenuates excessive insulin action on target cells. Therefore, RZ358 is ideally suited as a potential therapy for hyperinsulinism, and it is being developed to treat the hypoglycemia associated with diseases such as congenital HI. This is a Phase 2, multicenter, open label clinical study designed to assess the safety and efficacy of four progressively higher doses of RZ358 in separate groups of patients with hyperinsulinemic hypoglycemia due to Congenital HI, not adequately controlled with or without current standard of care. A screening period of up to 5 weeks will evaluate eligibility. Once enrolled, RZ358 will be administered bi-weekly over 8 weeks, and then patients will complete a post-treatment follow-up period of 13 weeks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
23

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Bulgaria
      • Sofia, Bulgaria
        • SHAT Children diseases "Prof. Dr. Ivan Mitov"
      • Varna, Bulgaria, 9010
        • Medical University of Varna UMHAT "St. Marina"
  • Canada
    • Qubec
      • Monteral, Qubec, Canada, H4A 3J1
        • Research Institute of the McGill University Health Centre
  • Denmark
      • Odense, Denmark, 5000
        • Odense University Hospital
  • Georgia
      • Tbilisi, Georgia, 0122
        • LTD "Pediatric Surgery Centre"
  • Germany
      • Magdeburg, Germany, 39120
        • Magdeburg University Clinic Center (Otto-von-Guericke Universität)
  • Israel
      • Jerusalem, Israel, 90000
        • Hadassah Har Hazofim MC - Division of Pediatric Endocrinology
    • Tel-Hashomer
      • Ramat Gan, Tel-Hashomer, Israel, 5265601
        • Edmond & Lilly Safra's Children Hospital
  • Russian Federation
      • Moscow, Russian Federation, 117036
        • Endocrinology Research Center
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d' Hebrón
  • Turkey
    • Adana
      • Sarıçam, Adana, Turkey
        • Adana Cukurova University Balcalı Hospital
    • Ankara
      • Çankaya, Ankara, Turkey, 06800
        • Hacettepe University
    • Diyarbakir
      • Kayapinar, Diyarbakir, Turkey, 21070
        • SBÜ Gazi Yaşargil Eğitim ve Araştirma Hastanesi
    • Erzurum
      • Yakutiye, Erzurum, Turkey
        • Erzurum City Hospital
  • United Kingdom
      • London, United Kingdom
        • Great Ormond Street Hospital
  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 41 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: RZ358 Cohort 1
Drug: RZ358 Sequential Group Cohort 1
IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks)
Experimental: RZ358 Cohort 2
Drug: RZ358 Sequential Group Cohort 2
IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks)
Experimental: RZ358 Cohort 3
Drug: RZ358 Sequential Group Cohort 3
IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks)
Experimental: RZ358 Cohort 4
Drug: RZ358 Sequential Group Cohort 4
IV infusion for 8 weeks (bi-weekly fixed dose-titration from 3 to 9 mg/kg for the first 4 weeks, followed by a fixed 9 mg/kg dose amount thereafter for the remaining 4 weeks)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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)
Time Frame: 8 weeks
The median of average daily percent time within the glucose target range 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline and End of Treatment (EOT) is reported.
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)
Time Frame: 8 weeks
The average daily percent time within the glucose target range (70-180 mg/dL) is compared from baseline to end of treatment (EOT) and the median percent change of that difference is reported.
8 weeks
Repeat Dose Pharmacokinetics of RZ358
Time Frame: 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
All patients who received RZ358 and for whom the primary PK data was considered to be sufficient and interpretable were to be included in the PK analyses. Individual and mean plasma concentration data is summarized descriptively at the specified timepoints. The results of this study may be combined with those of other studies for analysis and modeling (e.g., population PK and PK-PD), and therefore the PK parameters are reported separately, as part of an iterative population PK approach.
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

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT)
Time Frame: 8 weeks
The median of average weekly number of overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG at baseline (BL) and end of treatment (EOT) is reported.
8 weeks
Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL)
Time Frame: 8 weeks
The average weekly number overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported.
8 weeks
Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT)
Time Frame: 8 weeks
The median of average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia at baseline (BL) and end of treatment (EOT) is reported.
8 weeks
Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL)
Time Frame: 8 weeks
The average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported.
8 weeks
Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT)
Time Frame: 8 weeks
The median of average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM at baseline (BL) and end of treatment (EOT) is reported.
8 weeks
Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL)
Time Frame: 8 weeks
The average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported.
8 weeks
Occurrence of Hypoglycemia During Fasting Challenge
Time Frame: 8 Weeks
The occurrence of hypoglycemia during a 12 Hour fasting challenge.
8 Weeks
Median of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM at Baseline (BL) and End of Treatment (EOT)
Time Frame: 8 weeks
The median of average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM at baseline (BL) and end of treatment (EOT) is reported.
8 weeks
Median Net Change of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM From Baseline (BL)
Time Frame: 8 weeks
The average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM is compared from baseline (BL) to end of treatment (EOT) and the median net changes of those differences are reported.
8 weeks
Median of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM at Baseline (BL) and End of Treatment (EOT)
Time Frame: 8 weeks
The median of average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) at baseline (BL) and end of treatment (EOT) is reported.
8 weeks
Median Percent Change of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM From Baseline (BL)
Time Frame: 8 weeks
The average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) is compared from baseline (BL) to end of treeatment (EOT) and the median percent change of that difference is reported.
8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Rezolute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 24, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 5, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 19, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 21, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 31, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 4, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 28, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 9, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RZ358-606

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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