Study of RZ358-606 in Patients with Congenital hyperinsulinism
Research type
Research Study
Full title
An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism
IRAS ID
220725
Contact name
Indraneel Banerjee
Contact email
Sponsor organisation
XOMA (US) LLC
Eudract number
2016-004186-83
Duration of Study in the UK
1 years, 6 months, days
Research summary
Congenital hyperinsulinism (CHI) is the most frequent cause of severe, persistent hypoglycemia (low glucose, sugar, in blood) in newborn babies and children. The disease is characterised by a high blood level of insulin, leading to a decrease in glucose (sugar). Recurrent and severe hypoglycaemia can adversely affect brain function. Despite currently available therapies, CHI can result in serious neurological and developmental complications, including, recurrent seizures, learning disabilities, and focal brain lesions. The condition is currently managed by feeding the patients regular high carbohydrate meals and treating them with medicines to reduce insulin secretion, such as diazoxide, chlorothiazide, nifedipine, glucagon and octreotide. The treatment might include surgery of the pancreas (pancreatectomy). However, these treatments are difficult because these patients require constant monitoring and the drugs have undesirable side effects. Furthermore, although a rare disorder, CHI has been found to be a substantial economic burden on the NHS. XOMA 358 has been developed to treat hypoglycaemia associated with hyperinsulinemic (high level of insulin in blood) diseases such as CHI and post-gastric bypass hypoglycaemia. XOMA 358 binds to insulin receptors in order to sugar lowering effects of insulin on target cells.\nThe purpose of the study is to evaluate the safety and the effectiveness of XOMA 358 using multiple doses in patients from 2 years and above, suffering from hypoglycemia associated with CHI. Twenty patients are expected to take part in the study. The treatment consists of one weekly infusion of XOMA 358 for four weeks (induction). If the patient demonstrates a response to the study drug treatment without safety concerns, XOMA 358 therapy will be extended biweekly for 8 weeks. The patient then enters in a follow up period for 4 months. The approximate expected study duration for each patient is up to 8 months.\n
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
17/NW/0045
Date of REC Opinion
11 Apr 2017
REC opinion
Further Information Favourable Opinion