NCT05569746

A Study to Assess Safety, Efficacy, and Pharmacokinetics of INM004 in Pediatric Patients With STEC-HUS

Completed Phase 2 Last updated 21 August 2023

What this trial tests

Phase 2 trial testing INM004 in Diarrhea-Associated Hemolytic Uremic Syndrome in 57 participants. Completed in 14 July 2023.

Timeline

6 October 2022

Primary endpoint
15 May 2023

14 July 2023

Quick facts

Lead sponsor	Inmunova S.A.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	57
Start date	6 October 2022
Primary completion	15 May 2023
Estimated completion	14 July 2023
Sites	16 locations across Argentina

Drugs / interventions tested

• INM004 — full drug profile →
• SoC

Conditions studied

• Diarrhea-Associated Hemolytic Uremic Syndrome — all drugs for Diarrhea-Associated Hemolytic Uremic Syndrome →
• Pediatric Kidney Disease — all drugs for Pediatric Kidney Disease →
• Hemolytic-Uremic Syndrome — all drugs for Hemolytic-Uremic Syndrome →

Sponsor

Inmunova S.A. — full company profile →

Who can join

Adults 1 to 12, any sex, with Diarrhea-Associated Hemolytic Uremic Syndrome or Pediatric Kidney Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Hemolytic Uremic Syndrome (HUS) is a foodborne disease which mainly affects children. It is caused by Escherichia coli bacteria, which release a toxin called Shiga toxin within the body. This infectious form of HUS, defined as STEC-HUS, can cause sporadic cases or outbreaks, as observed in different countries. Argentina has the highest incidence of STEC-HUS worldwide. The disease is endemic, representing approximately 95% of all HUS cases nationwide.

STEC-HUS generally begins with diarrhea (with or without blood), and can also cause fever, abdominal pain, and cramps. Then the child may have pallor, altered consciousness, decreased urine output, seizures, and other symptoms. Although death is uncommon (it occurs in 2-4% of cases), it is a very serious disease that mainly affects the kidneys, and also other organs such as the brain. About half of children need to undergo a risky procedure such as dialysis (due to malfunctioning kidneys); and most of them also receive blood transfusions.

Around 30% of the patients are left with lifelong consequences that can range from permanent kidney damage to the need for a transplant.

So far there is no drug, antibiotic or vaccine to prevent or treat HUS. Current treatment protocols include hospitalization for all patients with HUS, and supportive therapy such as hydration and salt intake. Support therapy is not a specific treatment, but rather helps the body better defend itself against the disease.

The purpose of this study is to establish whether it is safe and effective to treat patients who are diagnosed with STEC-HUS, with INM004 (study drug). INM004 is an investigational product "Fraction F(ab')2 of Equine Shiga Antitoxin Immunoglobulin". It is a concentrated and sterile serum obtained from healthy horses immunized against Shiga toxin that contains antibodies capable of neutralizing it.

The initial hypothesis is that INM004 would neutralize the entry of Shiga toxin into the body's cells thus preventing the consequent toxic damage. With the proposed treatment, INM004 would eliminate the Shiga toxin, preventing the progression of HUS symptoms and its serious complications (such as the need for and duration of dialysis, duration of hospital stays, as well as neurological, cardiovascular, intestinal complications, among others) which are associated with high morbidity and mortality. This treatment could then have an impact in health costs of STEC-HUS as well as the social costs.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Open-label, controlled, phase 2 clinical trial assessing the safety, efficacy, and pharmacokinetics of INM004 in pediatric patients with Shiga toxin-producing Escherichia coli-associated hemolytic uremic syndrome.
   Fayad A, Principi I, Balestracci A, Alconcher L, et al · · 2025 · cited 6× · PMID 39528845 · DOI 10.1007/s00467-024-06583-3
2. Serology as an early diagnostic tool in pediatric patients with Shiga toxin-producing <i>Escherichia coli</i>-associated hemolytic uremic syndrome: a <i>post hoc</i> analysis of a phase 2 clinical trial.
   Landivar SM, Melli LJ, Pichel M, Rivas M, et al · · 2026 · PMID 41757982 · DOI 10.1128/jcm.01415-25