ZP4207-17103: Safety & Efficacy of Dasiglucagon in CHI patients
Research type
Research Study
Full title
A Randomized Trial in 2 parts: Double-Blind, Placebo-Controlled, Crossover Part 1 and Open-label Part 2, Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
IRAS ID
274234
Contact name
Indraneel (Indi) Banerjee
Contact email
Sponsor organisation
Zealand Pharma A/S
Eudract number
2017-004545-24
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 8 months, 0 days
Research summary
Summary of Research
Congenital hyperinsulinism (CHI) is a rare disorder in which β-cells in the pancreas secrete insulin irrespective of what the levels of glucose are in the plasma. This leads to persistent and severe lack of glucose in the bloodstream or, hypogylcaemia. CHI affects one in 50,000 newborns. Mutations in several different β-cell genes are known to cause CHI. Hypoglycaemia arising from CHI can lead to long-term neurological damage and impairment in neonates and infants. Up to 50% of children with CHI will experience neurodevelopmental abnormalities due to inadequate treatment and/or delay in diagnosis. Severe hypoglycaemia can result in epileptic seizures and coma. Major intellectual deficit is most frequent in children with neonatal initial onset whereas hypergylcaemia is less severe and brain damage is less frequent in children diagnosed with CHI later in childhood.
The ZP4207-17103 study is a 2-part randomised trial to evaluate the efficacy and safety of Dasiglucagon for the treatment of CHI in children.
Dasiglucagon is an investigational medicine which is a type of hormone that is produced by the human body.
In order to be eligible for the trial, potential participants have to be ≥7 days and <12 months of age at screening with a body weight of ≥2.0 kg (4.4 lbs.) and a diagnosis of CHI.The study consists of a screening period of 28 days, Part 1 is 2 X 48 hours long. Part 1 is double-blind and randomised, and placebo controlled. Part 2 of the study is an open-label active treatment period of 21 days. Part 4 is the follow-up period. The maximum trial duration for each patient is 81 days and maximum treatment period is 25 days.
The trial will take place at 5 to 7 sites in the US and Europe that are experienced in the treatment of CHI, recruiting approximately 12 patients.
Summary of Results
• Dasiglucagon was found to be efficacious in reducing glucose requirements in children with Congenital Hyperinsulinism (CHI).
o In Part 1 of the study, both primary and key secondary endpoints were met with statistically significant reductions in the mean Intraveneous Glucose Infusion Rate (IV GIR) during the last 12 hours and total amount of carbohydrates administered per day after dasiglucagon treatment compared to placebo.
o In Part 2 of the study, the majority of patients were weaned off GIR without increasing the time in hypoglycemia or the number of episodes, based on continuous glucose monitoring .
• Dasiglucagon was assessed to be safe and well tolerated and no new safety concerns were identified.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
20/YH/0001
Date of REC Opinion
25 Feb 2020
REC opinion
Further Information Favourable Opinion