Efficacy of INM004 in Children With STEC-HUS
This research study is about a new medicine called INM004, for children with a serious illness called Hemolytic-Uremic Syndrome (HUS). This type of HUS is caused by a germ called E. coli that produces a toxin (or poison). This illness can severely affect the kidneys and other organs. We are testing if INM004, given alongside standard care, can help children's kidneys recover better and faster. We also want to find out if it reduces serious problems, helps children leave hospital sooner, and is safe to use. Half the children will receive INM004 and half a dummy medicine (placebo) to fairly compare its effects.
At a glance
What is this study about?
This study is looking into a new medicine called INM004 for a serious condition known as Hemolytic-Uremic Syndrome (HUS), which affects children. Specifically, we're focusing on HUS caused by a type of E. coli bacteria that produces a harmful substance called Shiga toxin. This illness can lead to significant kidney damage, affect blood clotting, and cause other serious problems throughout the body.
The main goal is to see if INM004 can help children's kidneys recover better when they have this type of HUS. Researchers will also be checking if the medicine can reduce the risk of more severe complications, help children get better faster, and spend less time in hospital. We're also very keen to understand how safe INM004 is and how the body handles it.
To make sure we get clear answers, some children will receive INM004, and others will receive a placebo – a dummy medicine that looks the same but has no active ingredients. This allows doctors to fairly compare the results and determine if INM004 truly makes a difference beyond the usual care patients receive.
Key takeaways
- New medicine INM004 being tested for severe kidney condition (STEC-HUS) in children.
- Aims to improve kidney recovery and reduce serious problems.
- Some children get INM004, others a dummy medicine (placebo).
- Study includes regular checks and tests to monitor safety and effectiveness.
- Participation is voluntary and can be stopped at any time.
Who may be eligible?
This study is looking for children aged between 9 months and 17 years old. To be considered, the child must have recently developed HUS due to an E. coli infection that produces Shiga toxin. The specific E. coli infection needs to be confirmed through tests like stool samples or blood tests, and they must be a patient in the hospital where the study is taking place.
The doctors will also need to see clear signs of HUS, which includes problems with their kidneys, signs that their red blood cells are breaking down, and a low number of platelets (blood cells that help stop bleeding). For girls who have started their periods, a negative pregnancy test will be needed.
Children generally can't join if they started dialysis very recently before being admitted to hospital for this condition, or if too much time has passed since their HUS diagnosis at the hospital. Those with a history of certain long-term blood problems or kidney disease unrelated to HUS would also not be able to take part.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between 9 months and 17 years old?
- Has your child recently been diagnosed with HUS caused by a Shiga toxin-producing E. coli infection?
- Is your child currently admitted to the hospital where the study is taking place?
- Has your child shown signs of kidney damage, blood cell breakdown, and low platelets?
- Has your child NOT had chronic blood problems or kidney disease before this illness?
What does participation involve?
If your child takes part, they will either receive the new medicine, INM004, or a placebo (a dummy medicine) in addition to their usual hospital care. This will involve regular check-ups, blood tests, and possibly urine tests to see how they are responding to treatment and to monitor their recovery. The study doctors will carefully explain all procedures. We will also monitor how long your child stays in hospital and if any complications develop. The exact number of visits, tests, and total time involved will be fully explained by the study team before you decide to participate.
Potential risks and benefits
Locations (52)
- Hospital Interzonal Dr. José PennaVerified postcodeBahía Blanca, Argentina· Not yet recruiting
- Hospital PennaVerified postcodeBahía Blanca, Argentina· Not yet recruiting
- Hospital de niños Sor María LudovicaVerified postcodeLa Plata, Argentina· Recruiting
- Clinica del niño y la madreVerified postcodeMar del Plata, Argentina· Recruiting
- Hospital Interzonal Especializado Materno Infantil Don Victorio TetamantiVerified postcodeMar del Plata, Argentina· Recruiting
- Sanatorio de la Trinidad Ramos MejiaVerified postcodeRamos Mejía, Argentina· Not yet recruiting
- Hospital El CruceVerified postcodeSan Juan Bautista, Argentina· Not yet recruiting
- Sanatorio GüemesVerified postcodeBuenos Aires, Argentina· Active not recruiting
- Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan"Verified postcodeBuenos Aires, Argentina· Recruiting
- Hospital General de Niños Pedro de ElizaldeVerified postcodeBuenos Aires, Argentina· Active not recruiting
- Sanatorio AnchorenaUnverifiedCABA, Argentina· Active not recruiting
- Hospital Privado Centro Médico de CórdobaVerified postcodeCórdoba, Argentina· Recruiting
Common questions
What is STEC-HUS?
STEC-HUS is a serious kidney condition in children, caused by a type of E. coli bacteria that makes a harmful substance called Shiga toxin.
What is INM004?
INM004 is a new medicine being tested to see if it can help children's kidneys recover better from STEC-HUS.
Will my child definitely get the new medicine?
No, children in the study will either receive INM004 or a placebo (dummy medicine) to allow for a fair comparison of its effects.
How long will the study last for my child?
The exact duration will depend on your child's condition and recovery, but the study team will provide details of the expected timeframe during the consent process.
What if we change our mind about participating?
You are free to withdraw your child from the study at any point without it affecting their medical care.
How to find out more
Mariana Colonna, Bioch
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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