An extension study of Dasiglucagon in children with CHI
- Conditions
- Children with congenital hyperinsulinismMedDRA version: 20.0Level: SOCClassification code: 10027433Term: Metabolism and nutrition disorders Class: 6Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2024-514007-34-00
- Lead Sponsor
- Zealand Pharma A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 42
Completed treatment in either Trial ZP4207-17103 or ZP4207-17109, 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, Has a negative serum pregnancy test at baseline (only for females of child-bearing potential), 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. Only highly effective methods of birth control are accepted (i.e., one that results in less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices), or sexual abstinence.Only highly effective methods of birth control are accepted (i.e., one that results in less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices), or sexual abstinence, Able and willing to comply with trial procedures, 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.
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., 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term safety of dasiglucagon administered as SC infusion in children with CHI.;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;Primary end point(s): Adverse events
- Secondary Outcome Measures
Name Time Method Secondary end point(s):CGM percent time <70 mg/dL (3.9 mmol/L);Secondary end point(s):Rate of CGM-detected hypoglycemia episodes <70 mg/dL (3.9 mmol/L) for 15 minutes or more;Secondary end point(s):Rate of clinically significant CGM-detected hypoglycemia episodes <54 mg/dL (3.0 mmol/L) for 15 minutes or more;Secondary end point(s):Total amount of gastric carbohydrates administered (nasogastric [NG] tube or gastrostomy) to treat hypoglycemia;Secondary end point(s):Time to removal of NG tube or gastrostomy;Secondary end point(s):Time to pancreatic surgery (sub-total or total pancreatectomy)