Trial · NCT04538989
An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
NCT04538989, Phase 2, Completed, Rezolute; results first posted 2025-05-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism. Trials, record NCT04538989. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04538989
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
- NCT number
- NCT04538989
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 23 participants, actual
- Lead sponsor
- Rezolute
- Conditions
- Congenital Hyperinsulinism
- Interventions
- RZ358 Sequential Group Cohort 1, RZ358 Sequential Group Cohort 2, RZ358 Sequential Group Cohort 3, RZ358 Sequential Group Cohort 4
- First posted
- 2020-09-04
- Last updated
- 2025-05-28
- Results first posted
- 2025-05-28
- Limitations and caveats
Limitations include a small sample size (low statistical power), an open-label design (potential for bias due to lack of blinding), a short treatment period in a chronic disease, and a lack of ethnic diversity in study participants who have a rare pediatric disease. Additionally, the COVID-19 pandemic impacted the study by causing a delay in projected enrollment and study timelines. However, due to excess precaution taken, there was no impact on the overall execution/outcomes of the study.
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| 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) | 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. |
| 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) | 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. |
| Primary | Repeat Dose Pharmacokinetics of RZ358 | 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. |
| Secondary | Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT) | 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. |
| Secondary | Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL) | 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. |
| Secondary | Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | 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. |
| Secondary | Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | 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. |
| Secondary | Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | 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. |
| Secondary | Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | 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. |
| Secondary | Occurrence of Hypoglycemia During Fasting Challenge | 8 Weeks | The occurrence of hypoglycemia during a 12 Hour fasting challenge. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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) | 8 weeks | Per Protocol Population | Daily Percent Time | MEDIAN |
| 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) | 8 weeks | Per Protocol Population | Percent Change | MEDIAN |
| PRIMARY | Repeat Dose Pharmacokinetics of RZ358 | 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 | PK Population; Data from Cohort 1, 2 and 4 was not collected during Pre-dose Week 1. | ng/mL | MEAN |
| SECONDARY | Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT) | 8 weeks | Per Protocol Population; For efficacy outcomes, the PP Population included 20 participants for BGM-based glycemic endpoints due to endpoint-specific exclusions. One participant in Cohort 1 and one in Cohort 4 were excluded from the PP population BGM-based analyses because they failed to meet pre-specified minimum testing frequency by glucometer. One participant in Cohort 3 was also excluded from the PP population BGM analyses for stopping background SOC therapy after starting RZ358 in the study | Hypoglycemia Events | MEDIAN |
| SECONDARY | Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL) | 8 weeks | Per Protocol Population; For efficacy outcomes, the PP Population included 20 participants for BGM-based glycemic endpoints due to endpoint-specific exclusions. One participant in Cohort 1 and one in Cohort 4 were excluded from the PP population BGM-based analyses because they failed to meet pre-specified minimum testing frequency by glucometer. One participant in Cohort 3 was also excluded from the PP population BGM analyses for stopping background SOC therapy after starting RZ358 in the study | Percent Change | MEDIAN |
| SECONDARY | Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | 8 weeks | Per Protocol Population | Daily Percent Time | MEDIAN |
| SECONDARY | Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | 8 weeks | Per Protocol Population | Percent Change | MEDIAN |
| SECONDARY | Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | 8 weeks | Per Protocol Population | Daily Minutes in Hypoglycemia | MEDIAN |
| SECONDARY | Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | 8 weeks | Per Protocol Population | Percent Change | MEDIAN |
| SECONDARY | Occurrence of Hypoglycemia During Fasting Challenge | 8 Weeks | Per Protocol Population | participants | NUMBER |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group267
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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) | RZ358 Cohort 1 | Median of Average Daily Percent Time in 70-180 mg/dL at BL | 81.7 | 95% Confidence Interval 70.5 to 92.5 | Daily Percent Time |
| 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) | RZ358 Cohort 2 | Median of Average Daily Percent Time in 70-180 mg/dL at BL | 72.9 | 95% Confidence Interval 67.5 to 80.9 | Daily Percent Time |
| 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) | RZ358 Cohort 3 | Median of Average Daily Percent Time in 70-180 mg/dL at BL | 83.4 | 95% Confidence Interval 43.2 to 94.8 | Daily Percent Time |
| 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) | RZ358 Cohort 4 | Median of Average Daily Percent Time in 70-180 mg/dL at BL | 59.3 | 95% Confidence Interval 25.7 to 110 | Daily Percent Time |
| 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) | RZ358 Pooled | Median of Average Daily Percent Time in 70-180 mg/dL at BL | 76.9 | 95% Confidence Interval 65.3 to 80.7 | Daily Percent Time |
| 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) | RZ358 Cohort 1 | Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) | 84.8 | 95% Confidence Interval 71.6 to 100.3 | Daily Percent Time |
| 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) | RZ358 Cohort 2 | Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) | 78.5 | 95% Confidence Interval 61.1 to 88.6 | Daily Percent Time |
| 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) | RZ358 Cohort 3 | Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) | 82.9 | 95% Confidence Interval 67.1 to 91.6 | Daily Percent Time |
| 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) | RZ358 Cohort 4 | Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) | 70.8 | 95% Confidence Interval 50.9 to 93.9 | Daily Percent Time |
| 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) | RZ358 Pooled | Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) | 79.1 | 95% Confidence Interval 72.0 to 83.9 | Daily Percent Time |
1 to 10 of 267Source ClinicalTrials.gov
See all 267 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RZ358 Cohort 1 | 4 | |
| Overall Study | STARTED | RZ358 Cohort 2 | 8 | |
| Overall Study | STARTED | RZ358 Cohort 3 | 8 | |
| Overall Study | STARTED | RZ358 Cohort 4 | 3 | |
| Overall Study | COMPLETED | RZ358 Cohort 1 | 4 | |
| Overall Study | COMPLETED | RZ358 Cohort 2 | 8 | |
| Overall Study | COMPLETED | RZ358 Cohort 3 | 8 | |
| Overall Study | COMPLETED | RZ358 Cohort 4 | 3 | |
| Overall Study | NOT COMPLETED | RZ358 Cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | RZ358 Cohort 2 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events160
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Phlebitis | Vascular disorders | RZ358 Cohort 1 | 0 | 4 | 0 |
| other | Phlebitis | Vascular disorders | RZ358 Cohort 2 | 1 | 8 | 1 |
| other | Phlebitis | Vascular disorders | RZ358 Cohort 3 | 0 | 8 | 0 |
| other | Phlebitis | Vascular disorders | RZ358 Cohort 4 | 1 | 3 | 1 |
| other | Extravasation | General disorders | RZ358 Cohort 1 | 0 | 4 | 0 |
| other | Extravasation | General disorders | RZ358 Cohort 2 | 1 | 8 | 1 |
| other | Extravasation | General disorders | RZ358 Cohort 3 | 0 | 8 | 0 |
| other | Extravasation | General disorders | RZ358 Cohort 4 | 0 | 3 | 0 |
| other | Fatigue | General disorders | RZ358 Cohort 1 | 0 | 4 | 0 |
| other | Fatigue | General disorders | RZ358 Cohort 2 | 1 | 8 | 1 |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RZ358 Cohort 1 | 0 | 4 |
| Total, all-cause mortality | RZ358 Cohort 2 | 0 | 8 |
| Total, all-cause mortality | RZ358 Cohort 3 | 0 | 8 |
| Total, all-cause mortality | RZ358 Cohort 4 | 0 | 3 |
| Total, other adverse events | RZ358 Cohort 1 | 2 | 4 |
| Total, other adverse events | RZ358 Cohort 2 | 7 | 8 |
| Total, other adverse events | RZ358 Cohort 3 | 5 | 8 |
| Total, other adverse events | RZ358 Cohort 4 | 2 | 3 |
| Total, serious adverse events | RZ358 Cohort 1 | 0 | 4 |
| Total, serious adverse events | RZ358 Cohort 2 | 2 | 8 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40107271 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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