ZP4207-17109 open-label study of Dasiglucagon in children with CHI
Research type
Research Study
Full title
A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
IRAS ID
248535
Contact name
Indraneel (Indi) Banerjee
Contact email
Sponsor organisation
Zealand Pharma A/S
Eudract number
2017-004547-21
Clinicaltrials.gov Identifier
135869, IND
Duration of Study in the UK
1 years, 4 months, 29 days
Research summary
Congenital hyperinsulinism (CHI) is a rare and challenging disorder in which excessive insulin secretion by pancreas causes repeated and often severe hypoglycaemia (low blood sugar). Hypoglycaemia is the main risk of CHI as in severe cases can cause long-term neurological impairments. \nThe ZP4207-17109 study is a two treatment period study to evaluate the efficacy and safety of Dasiglucagon in children with CHI. Dasiglucagon is an investigational medicine (not yet approved by regulatory authorities) which is similar to glucagon, a type of hormone produced by the human body. Dasiglucagon has a form of solution and will be administered through an infusion pump.\nIf the study medicine works as expected, it may help to reduce the amount and frequency of nutritional feeds patients with CHI requires, while avoiding hypoglycaemia, which could substantially reduce the disease burden in this patients.Eligible patients will be age 3 months to 12 years with confirmed diagnose of CHI, who meet all of study eligibility criteria.\nThe study consists of 4 phases: Screening (at least 14 days up to 28 days), Treatment Period 1 (4 weeks), Treatment Period 2 (4 weeks) and follow up (4 weeks). During the treatment period 1 patients will be randomly (by chance) assigned to 1 of 2 treatment groups:\n1.Standard of Care (SoC) treatment, or\n2.\tStandard of Care plus Dasiglucagon treatment. During the treatment period 2 all patients will receive Standard of Care plus Dasiglucagon treatment. At the study doctor’s discretion patients may be discharged from the hospital to continue treatment at home.\nPatients’ participation in the study will last for an approximately 16 weeks. The study medicine treatment duration will be 4 or 8 weeks, depending of treatment group assignment.\nPatients will make at a minimum a total of 8 planned visits to the study centre. Additional visits during the screening period, to complete all required procedures, or during the treatment period, may be required.During the study patients will need to be hospitalised before receiving the study medicine and will undergo assessments, which will include blood tests, electrocardiogram (ECG), echocardiogram, fasting glucose tolerance test and blood sugar monitoring.\nApproximately 32 children from up to 11 study centres in UK, Germany and USA will take part in this study. [COVID-19 amendment 16/04/2020] Due to COVID-19, the changes made to the trial are considered as ’Urgent Safety Measures’, therefore implemented with immediate effect and without prior approvals from the National Competent Authorites or Ethics Committees. The measures are only implemented during the exceptional situation due to COVID-19 and will be reverted as soon as the situation allows.\nThe changes will comprise:\nFor patients, who are in the screening phase in ZP4207-17109:\nIf needed, sites can postpone the randomization visit and prolong the screening period with a maximum of 2 months. If the screening period is extended with more than a month, a new safety lab sample should be collected and evaluated before randomization.\nFor patients who are in the treatment phase (dasiglucagon or standard of care):\n- Visit 3, 4 and 6 may be converted to phone (or video) visits. Visit 1, 2, 5 and 7 have to be performed on site. The visit window for visit 5 may be extended with +7 days. If a patient is qualifying for visit 8, this can be converted to a phone (or video) visit.\n- At the site visits, all assessments should be performed to the extent possible. If local restrictions apply for safety measurements (e.g., restricted availability of lab services, or echocardiography), the decision on which measurements to be performed will be at the investigator’s discretion.\n- At the dispensing visits, sites should dispense the usual kits and consider to dispense additional kits for visits that could be converted to phone visits.\n- If the site has the infrastructure to deliver the IMP to the patients’ home this can be done in accordance with the procedure specified by the Sponsor or the hospital’s regulations.\nFor sites that are not permitting monitoring visits:\nMonitoring visits should be converted to phone visits\nRemote monitoring will be used to the extent possible\nAn information sheet for parents/participants including the changes made to the study due to COVID-19 is also included.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
18/YH/0264
Date of REC Opinion
24 Aug 2018
REC opinion
Further Information Favourable Opinion