Phase III study of INM004 in pediatric patients with HUS
Research type
Research Study
Full title
A Phase III study to evaluate the efficacy of INM004 (Shiga antitoxin) in pediatric patients with Shiga toxin-producing Escherichia coli-associated Hemolytic Uremic Syndrome.
IRAS ID
1010652
Contact name
Enrico Colli
Contact email
Sponsor organisation
Chemo Research S.L.
Clinicaltrials.gov Identifier
Research summary
If people get a gut infection with a bacteria called E.coli, they usually have a few days of diarrhoea and vomiting, then get better.Some types of E.coli can make a particular toxin, called Shiga toxin, which is very poisonous to parts of the body.This toxin can cause breakdown of blood cells (called haemolysis) and build-up of body wastes (called uraemia) by affecting how well the kidneys work.This is called Shiga toxin E.coli – Haemolytic Uraemic Syndrome, STEC-HUS.Some people, particularly children and the elderly, are at greater risk of STEC-HUS and may need dialysis to support the kidneys until they get better (and in a small number of people, this does not happen and patients are left with kidney failure).Currently there is no specific treatment for STEC-HUS proven to be effective.Treatment includes managing fluids and electrolytes, and dialysis if needed. This study is to work out whether a new treatment, INM004, can be used during the early (acute) part of the STEC-HUS illness.INM004 is an antibody (a type of protein usually made by the body’s immune system in response to an infection) which specifically affects the Shiga toxin, stopping it from acting once it is in the bloodstream.The hope is that by stopping the toxin early, it does not have chance to cause as much damage to kidneys and other organs.The study will look at whether giving INM004 will help the kidneys get better faster, and maybe either avoid the need for dialysis or need dialysis for less time – we already know that needing dialysis for STEC-HUS can be associated with later kidney disease, and need for a kidney transplant.As children are at highest risk of these complications, the study will be done in children aged 9 months to <18 years.The study will be done in hospitals with specialist children’s kidney teams in Argentina, the EU, and the UK, and will run for 2 years.Patients in the study will receive either 2 doses of INM00 or a placebo 24 hours apart, through an intravenous line.
REC name
Wales REC 5
REC reference
24/WA/0275
Date of REC Opinion
13 Nov 2024
REC opinion
Further Information Favourable Opinion