Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as RZ358 Sequential Group Cohort 1
RZ358 Sequential Group · 1 trial · 1 indication
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.
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.
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.
| Arm | Type | Description |
|---|---|---|
| RZ358 Cohort 1 | EXPERIMENTAL | - |
| RZ358 Cohort 2 | EXPERIMENTAL | - |
| RZ358 Cohort 3 | EXPERIMENTAL | - |
| RZ358 Cohort 4 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| RZ358 Sequential Group Cohort 1 | DRUG | IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks) |
| RZ358 Sequential Group Cohort 2 | DRUG | IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks) |
| RZ358 Sequential Group Cohort 3 | DRUG | IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) |
| RZ358 Sequential Group Cohort 4 | DRUG | 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) |
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, duri...