Children with congenital hyperinsulinism MedDRA version: 20.0 Level: PT Classification code 10061211 Term: Hyperinsulinism System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Product Name: Dasiglucagon
Product Code: ZP4207
Pharmaceutical Form: Solution for injection in administration system
INN or Proposed INN: Dasiglucagon
CAS Number: 1544300-84-6
Current Sponsor code: ZP
Sponsors
Zealand Pharma A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Established and documented diagnosis of CHI based on standard of care. 2. Male or female between 3 months and 12 years of age (both inclusive) at screening. 3. Has a negative serum pregnancy test at screening/baseline (only for girls of childbearing potential). 4. Sexually active female patients (and their partners) must continue to use acceptable contraception or refrain from sexual activity from screening until 30 days after the last dose of trial drug. Females must abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception. Abstinence can only be accepted if this is true abstinence in line with the preferred and usual lifestyle of the patient. Acceptable methods of contraception are: a) Hormonal contraceptives (e.g., oral contraceptive pill, depot, patch, intramuscular implant or injection, sponge, or vaginal ring), stabilized for at least 30 days if first use or b) Barrier method, e.g., (i) condom (male or female) and (ii) diaphragm with spermicide 5. Experiencing =3 events of hypoglycaemia per week (PG =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Previous administration of dasiglucagon (previously referred to as ZP4207). 2. Known or suspected allergy to the trial drug or related products. 3. Previous participation (randomization) in this trial. 4. Circulatory instability requiring supportive medication or presence of hypertension or hypotension, including circulatory instability requiring supportive medication or presence of pheochromocytoma. 5. Requires exogenous insulin. 6. Body weight of 20 mg/mg/m2 body surface area or equivalent in the 5 days before screening. 10. Use of anti-inflammatory biological agents, or other immune-modulating agents in the 3 months prior to screening. 11. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5 X the upper limit of normal (ULN), or estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2 adjusted by a pediatric formula (e.g., Schwartz formula). 12. Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the patient’s ability to participate in the trial. 13. History of laboratory test results obtained before screening that show presence of HIV, hepatitis B surface antigen, hepatitis C antibody, or hepatitis A immunoglobulin M. 14. Any recognized clotting or bleeding disorders. 15. Has participated in an interventional clinical trial (investigational or marketed product) within 3 months of screening or 5 half-lives of the drug under investigation (whichever comes first), or plans to participate in another clinical trial. 16. The use of prescription or non-prescription medications known to cause QT prolongation. Randomization Exclusion Criteria: 1. Use of glucagon within 24 hours before randomization. 2. Significant changes to CHI medications during screening. 3. An average of <3 events per week of hypoglycemia, as recorded in the diary during the 2 weeks prior to randomization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Weeks 2-4;Main Objective: To evaluate the efficacy of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with Congenital Hyperinsulinism.;Secondary Objective: -To evaluate the safety and tolerability of dasiglucagon administered as an SC infusion in children with CHI -To evaluate the efficacy of dasiglucagon in reducing glucose requirements -To investigate quality of life and resource utilization;Primary end point(s): Treatment Period 1: Hypoglycaemia event rate, defined as average weekly number of hypoglycaemic events (PG <70 mg/dL or 3.9 mmol/L) during Weeks 2-4, as detected by self-monitored PG (SMPG). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Treatment Period 1 - Increase in fasting tolerance (time from beginning of meal to the beginning of the first continuous 15-minute Continuous glucose monitoring [CGM] reading <70 mg/dL [3.9 mmol/L]. - CGM percent time in range 70-180 mg/dL (3.9-10.0 mmol/L) during Weeks 2-4. - Clinically significant hypoglycemia event rates, defined as average weekly number of events < 54 mg/dL (3.0 mmol/L) as detected by SMPG during Weeks 2-4.;Timepoint(s) of evaluation of this end point: Weeks 2-4 | — |
Countries
Germany, Israel, United Kingdom, United States
Contacts
Public ContactClinical Trial Manager
Zealand Pharma A/S
Outcome results
None listed