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
Sponsors
Zealand Pharma A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Completed treatment in either Trial ZP4207-17103 or ZP4207-17109 2. Expected to continue to have a positive benefit-risk assessment for treatment with dasiglucagon (based on considerations of glycemic effect, tolerability, and nature and frequency of AEs experienced in the lead-in trial), with signed investigator statement documenting the positive benefit-risk assessment made 3. Has a negative serum pregnancy test at baseline (only for females of child-bearing potential) 4. Sexually active female patients and their partners must use acceptable contraception or refrain from sexual activity from baseline 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. Able and willing to comply with trial procedures 6. Following receipt of oral and written information about the trial, the patient (depending on local institutional review board [IRB]/independent ethics committee [IEC] requirements) must provide assent and one or both parents* or guardian of the patient must provide signed informed consent before any trial-related activity is carried out. * If required by local regulations, both parents must give their permission unless one parent is deceased, unknown, incompetent, or not reasonably available, or when only one parent has legal responsibility for the care and custody of the child. Are the trial subjects under 18? yes Number of subjects for this age range: 44 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. The patient developed any conditions prohibited by the lead-in trial, requires medication prohibited by the lead-in trial, or has other new complications that preclude participation in the investigator’s opinion. 2. Has participated in an interventional clinical trial (investigational or marketed product) within 3 months before baseline or 5 half-lives of the drug under investigation (whichever comes first) or plans to participate in another clinical trial. Excluded from this is participation in Trial ZP4207-17103, Trial ZP4207-17109, and/or 18F-Dopa positron emission tomography computed tomography/magnetic resonance imaging investigation (when performed as a part of a clinical trial) for diagnosis of focal CHI.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety of dasiglucagon administered as SC infusion in children with CHI.;Primary end point(s): - Adverse events;Timepoint(s) of evaluation of this end point: each 3 month period for the first year; subsequent years will have longer periods assigned for analysis.; Secondary Objective: -To evaluate the long-term efficacy of dasiglucagon in reducing hypoglycemia -To evaluate the long-term efficacy of dasiglucagon in reducing glucose requirements -To evaluate the long-term tolerability of dasiglucagon administered as SC infusion in children with CHI -To investigate quality of life (QoL) and resource utilization | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Total amount of gastric carbohydrates administered (nasogastric [NG] tube or gastrostomy) to treat hypoglycemia -Time to removal of NG tube or gastrostomy -Time to pancreatic surgery (sub-total or total pancreatectomy) -Continuous glucose monitoring (CGM) percent time below 70 mg/dL (3.9 mmol/L) -Number of hypoglycemic events (PG <70 mg/dL [3.9 mmol/L]) as detected by self-monitored plasma glucose (SMPG) -Incidence of clinically significant episodes of hypoglycemia, defined as <54 mg/dL (3.0 mmol/L) for 15 minutes or more, as measured by CGM ;Timepoint(s) of evaluation of this end point: each 3 month period for the first year; subsequent years will have longer periods assigned for analysis. | — |
Countries
Germany, Israel, United Kingdom, United States
Contacts
Public ContactClinical Trial Manager
Zealand Pharma A/S
Outcome results
None listed