A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients with Congenital Hyperinsulinism

Conditions

Hypoglycemia associated with congenital hyperinsulinism

Brief summary

Change in average weekly incidence of hypoglycemia events (<70 mg/dL or <3.9 mmol/L) by SMBG after 24 weeks.

Detailed description

Change in average daily percent time in hypoglycemia (<70 mg/dL or <3.9 mmol/L) by CGM after 24 weeks., Safety and tolerability of RZ358 based on assessments of AEs, SAEs, clinical laboratory measurements, ECG, vital signs, physical examination, immunogenicity assessments, hepatic ultrasound, and incidence of hyperglycemia events by SMBG at thresholds of >250 mg/dL and >300 mg/dL (>13.9 mmol/L and >16.7 mmol/L, respectively.), a. -Change in average weekly incidence of potentially serious hypoglycemia events by SMBG -Change in average daily percent time with potentially serious hypoglycemia by CGM -Change in average 8-hour overnight percent time with hypoglycemia by CGM, b. -Change in average daily duration (min) with hypoglycemia by CGM -Proportion of participants achieving <4% average daily time in hypoglycemia by CGM -Proportion of participants experiencing no potentially serious hypoglycemia events by SMBG, The repeat-dose PK profile of RZ358 assessed by popPK modeling, which will be used to estimate PK parameters by dose and age category.

Related papers

No related papers found yet.

Interventions

DRUGRZ358 placebo is a sterile solution for parenteral administration with a minimum extractable volume of 1.0 mL in an ISO 2R

DRUGtype I clear glass vial with a fluoropolymer-faced

DRUGgrey chlorobutyl rubber stopper

DRUGand an aluminum seal with plastic flip-off cap. The buffered vehicle nominal composition is as follows: 10mM L-histidine

DRUG10mM L- methionine

DRUG270mM sorbitol

DRUG0.01% polysorbate 20

DRUGpH 5.8.

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Change in average weekly incidence of hypoglycemia events (<70 mg/dL or <3.9 mmol/L) by SMBG after 24 weeks.	—

Secondary

Measure	Time frame
Change in average daily percent time in hypoglycemia (<70 mg/dL or <3.9 mmol/L) by CGM after 24 weeks., Safety and tolerability of RZ358 based on assessments of AEs, SAEs, clinical laboratory measurements, ECG, vital signs, physical examination, immunogenicity assessments, hepatic ultrasound, and incidence of hyperglycemia events by SMBG at thresholds of >250 mg/dL and >300 mg/dL (>13.9 mmol/L and >16.7 mmol/L, respectively.), a. -Change in average weekly incidence of potentially serious hypoglycemia events by SMBG -Change in average daily percent time with potentially serious hypoglycemia by CGM -Change in average 8-hour overnight percent time with hypoglycemia by CGM, b. -Change in average daily duration (min) with hypoglycemia by CGM -Proportion of participants achieving <4% average daily time in hypoglycemia by CGM -Proportion of participants experiencing no potentially serious hypoglycemia events by SMBG, The repeat-dose PK profile of RZ358 assessed by popPK modeling, which will b	—

Measure

Time frame

Change in average daily percent time in hypoglycemia (<70 mg/dL or <3.9 mmol/L) by CGM after 24 weeks., Safety and tolerability of RZ358 based on assessments of AEs, SAEs, clinical laboratory measurements, ECG, vital signs, physical examination, immunogenicity assessments, hepatic ultrasound, and incidence of hyperglycemia events by SMBG at thresholds of >250 mg/dL and >300 mg/dL (>13.9 mmol/L and >16.7 mmol/L, respectively.), a. -Change in average weekly incidence of potentially serious hypoglycemia events by SMBG -Change in average daily percent time with potentially serious hypoglycemia by CGM -Change in average 8-hour overnight percent time with hypoglycemia by CGM, b. -Change in average daily duration (min) with hypoglycemia by CGM -Proportion of participants achieving <4% average daily time in hypoglycemia by CGM -Proportion of participants experiencing no potentially serious hypoglycemia events by SMBG, The repeat-dose PK profile of RZ358 assessed by popPK modeling, which will b

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Countries

Bulgaria, Denmark, France, Germany, Greece, Spain

Outcome results

None listed