Phase 2
N=23
An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Bottom Line
View on ClinicalTrials.gov: NCT04538989 ↗Enrolled (actual)
23
Serious AEs
13.0%
Results posted
May 2025
Primary outcome: Primary: 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) — 81.7; 72.9; 83.4; 59.3 Daily Percent Time
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- RZ358 Sequential Group Cohort 1 (Drug); RZ358 Sequential Group Cohort 2 (Drug); RZ358 Sequential Group Cohort 3 (Drug); RZ358 Sequential Group Cohort 4 (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Rezolute
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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) |
81.7; 72.9; 83.4; 59.3; 76.9; 84.8 | — |
| PRIMARY 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) |
6.19; 0.31; 5.52; 9.07; 4.59 | — |
| PRIMARY Repeat Dose Pharmacokinetics of RZ358 |
55648; 55648; 29093; 120368; 164047; 43593 | — |
| SECONDARY Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT) |
10.1; 12.5; 13.0; 9.30; 11.5; 9.92 | — |
| SECONDARY Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL) |
-1.6; -47.7; -84.4; -19.9; -58.9; 14.6 | — |
| SECONDARY Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) |
16.3; 20.3; 13.7; 34.4; 16.2; 12.3 | — |
| SECONDARY Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) |
-31.1; -64.6; -61.0; -46.6; -53.5; -19.3 | — |
| SECONDARY Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) |
204; 266; 180; 472; 214; 158 | — |
| SECONDARY Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) |
-22.2; -63.7; -57.5; -40.2; -49.1; 9.19 | — |
| SECONDARY Occurrence of Hypoglycemia During Fasting Challenge |
3; 2; 0; 0; 5; 3 | — |
| SECONDARY Median of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM at Baseline (BL) and End of Treatment (EOT) |
3.00; 3.50; 2.46; 4.47; 3.37; 2.37 | — |
| SECONDARY Median Net Change of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM From Baseline (BL) |
-0.47; -1.41; -1.49; -1.14; -1.05; -0.32 | — |
| SECONDARY 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) |
80.8; 75.0; 82.1; 48.0; 75.0; 84.4 | — |
| SECONDARY Median Percent Change of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM From Baseline (BL) |
7.59; 3.67; -2.64; 44.1; 4.22 | — |
Summary
The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04538989). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.