Treatment of Hemolytic Uremic Syndrome in Children with INM004

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the efficacy, safety, and pharmacokinetics of INM004 in pediatric patients suffering from Hemolytic Uremic Syndrome (STEC-HUS) caused by Shiga toxin-producing Escherichia coli. The primary goal is to assess how well INM004, when added to standard care, improves renal function. Secondary objectives include reducing mortality, preventing extrarenal complications, and shortening hospital stays. The study involves a randomized, controlled design comparing INM004 to a placebo.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Age ≥ 9 months and \< 18 years at the time of randomization.
2. In addition, only for subjects \< 1 year and ≥ 15 years, confirmation of STEC infection determined by:

   1. Detection of generic Stx, Stx1, Stx2, or Stx1/Stx2 in stools by enzyme immunoassay (EIA); or
   2. Detection of stx, stx1, stx2, or stx1/stx2 genes in stools by Polymerase Chain Reaction (PCR); or
   3. Detection of specific anti-polysaccharide (IgM) antibodies in serum; or
   4. Fecal culture positive for E. coli O157 confirmed by serogroup-specific seroagglutination.
3. Hospitalization at the participating institution.
4. History of onset of diarrhea within 10 days prior to STEC-HUS diagnosis at the participating institution.
5. Diagnosis of STEC-HUS defined as a subject with signs of renal damage, hemolysis, and platelet consumption:

   1. Signs of renal damage defined as:

      * Serum creatinine value above the ULN for age and sex, and GFR below the LLN for age, sex, and height.
   2. Presence of hemolysis documented by:

      * LDH levels above the ULN for age, and/or
      * Presence of schistocytes in peripheral blood smear.
   3. Platelet consumption according to any of the following laboratory criteria:

      * Peripheral blood platelet count \< 150 × 103/μL, and/or
      * A ≥50% decrease in peripheral blood platelet count compared to a sample collected within the previous 24 hours.
6. Informed consent form signed and dated by the subject or, the legal guardian(s), with the subject's assent as appropriate based on age and regulatory guidelines in the region.
7. Subjects who have already had menarche must have a negative pregnancy test.

Exclusion Criteria:

1. Start of dialysis within 48 hours prior to admission to the participating institution.
2. More than 24 hours from diagnosis of STEC-HUS at the participating institution up to randomization.
3. History of chronic/recurrent hemolytic anemia, thrombocytopenia, or CKD.
4. Personal and/or family history of atypical HUS.
5. Suspected HUS secondary to infectious processes other than gastrointestinal (e.g., Streptococcus pneumoniae, HIV).
6. Suspected HUS secondary to other etiologies (e.g., drug-associated HUS, neoplasms, bone marrow or solid organ transplantation, autoimmune disorders).
7. Any other acute or chronic medical condition that, in the opinion of the investigator, may interfere with the evaluation of the efficacy and/or safety of the study medication.
8. History of: a) anaphylaxis of any kind; b) prior administration of equine serum (e.g., antivenom, anti-arachnid serum, anti-SARS-CoV-2 serum, etc.) or an allergic reaction from contact or exposure to horses.
9. Pregnant or breastfeeding woman.
10. Impossibility of hospitalization in the participating institution.
11. Concurrent participation in another clinical trial or having participated in a clinical trial in the last 3 months.
12. Severe malnutrition. Defined when the weight is three standard deviations below the median, according to height, age and sex as per WHO guidelines.
13. Medical conditions that may affect kidney function or cause/enhance neurological symptoms or signs:

    * Congenital or acquired anomalies that may affect functioning renal mass.
    * Epilepsy or structural abnormalities of the brain that may increase the risk of seizures.
    * Trisomy 21.
    * Prematurity (born before 28 weeks gestation).
    * Other (according to investigator criteria).

Where it's running

• Hospital de niños Sor María Ludovica
  La Plata, Buenos Aires, Argentina Recruiting
• Clinica del niño y la madre
  Mar del Plata, Buenos Aires, Argentina Recruiting
• Hospital Interzonal Especializado Materno Infantil Don Victorio Tetamanti
  Mar del Plata, Buenos Aires, Argentina Recruiting
• Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan"
  Buenos Aires, Buenos Aires F.D., Argentina Recruiting
• Hospital Privado Centro Médico de Córdoba
  Córdoba, Córdoba Province, Argentina Recruiting
• Hospital de Niños de la Santísima Trinidad
  Córdoba, Córdoba Province, Argentina Recruiting

+2 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Mariana Colonna, Bioch
• Email: mcolonna@inmunova.com
• Phone: +541120331455

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.