Efficacy of INM004 in Children With STEC-HUS

NCT ID: NCT06389474

Last Updated: 2026-01-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

220 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-05

Study Completion Date

2026-12-31

Brief Summary

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The objectives of this study are to evaluate the efficacy, safety, and pharmacokinetics of INM004 in pediatric patients with Hemolytic Uremic Syndrome associated to infection by Shiga toxin-producing Escherichia coli (STEC-HUS).

Detailed Description

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The primary objective will be to evaluate the efficacy of INM004, added to the standard of care, as a treatment for STEC-HUS in the amelioration of renal function.

Secondary objectives

* To evaluate the efficacy of INM004 in the reduction of mortality.
* To evaluate the efficacy of INM004 in the prevention and reduction of extrarenal complications.
* To evaluate the efficacy of INM004 in the improvement of TMA laboratory parameters.
* To evaluate the efficacy of INM004 in the reduction of hospital stay days.
* To evaluate the safety of INM004
* To evaluate the pharmacokinetics of INM004
* To evaluate the kinetics of Stx

Conditions

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Hemolytic-Uremic Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Adaptive design. IA will be done when approximately 50% of the enrollment is reached and the participants have completed 28 days of follow-up. This analysis will not be blinded and will be carried out to declare futility or re-estimate the sample size. The re-estimation of the sample size will allow a maximum of 300 randomized subjects in total, but a reduction in the initial item size of 220 subjects is not expected. In case of re-estimating the sample size, the smallest sample size under 300 will be selected, which allows a power of ≥ 80%. %. If with 300 subjects, a power of 80% is not reached, but it is ≥ 50%, then the sample size is re-estimated to 300 subjects, as long as the conditional power of the IA is ≥ 50% (promising results)
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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INM004

Two doses of Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin F(ab´)2 fragment at a dosage of 4 mg/kg of body weight, 24 hours apart.

Group Type EXPERIMENTAL

INM004

Intervention Type BIOLOGICAL

Two doses of Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin F(ab´)2 fragments at a dosage of 4 mg/kg of body weight, 24 hours apart. Each vial contains 25 mg of protein/ml. Therefore, each subject must receive 0.16 ml/kg per dose. The vial volume will be reconstituted in a 100 ml (for subjects with a body weight of 20 kg or more) or 50 ml (for subjects under 20 kg of body weight) infusion bag. It will be administered as an intravenous infusion using an infusion pump over a period of 50 minutes. In subjects with a BMI over 30 kg/m2, infusion will be performed over a period of 100 minutes

Placebo

Two doses of saline solution, 24 hours apart.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Two doses of placebo, 24 hours apart. The placebo solution has the same composition of excipients as INM004 without the active pharmaceutical ingredient, and its appearance is identical. Each subject must receive 0.16 ml/kg of placebo solution per dose. The vial volume will be reconstituted in a 100 ml (for subjects with a body weight of 20 kg or more) or 50 ml (for subjects under 20 kg of body weight) infusion bag. It will be administered as an intravenous infusion using an infusion pump over a period of 50 minutes. In subjects with a BMI over 30 kg/m2, infusion will be performed over a period of 100 minutes

Interventions

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INM004

Two doses of Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin F(ab´)2 fragments at a dosage of 4 mg/kg of body weight, 24 hours apart. Each vial contains 25 mg of protein/ml. Therefore, each subject must receive 0.16 ml/kg per dose. The vial volume will be reconstituted in a 100 ml (for subjects with a body weight of 20 kg or more) or 50 ml (for subjects under 20 kg of body weight) infusion bag. It will be administered as an intravenous infusion using an infusion pump over a period of 50 minutes. In subjects with a BMI over 30 kg/m2, infusion will be performed over a period of 100 minutes

Intervention Type BIOLOGICAL

Placebo

Two doses of placebo, 24 hours apart. The placebo solution has the same composition of excipients as INM004 without the active pharmaceutical ingredient, and its appearance is identical. Each subject must receive 0.16 ml/kg of placebo solution per dose. The vial volume will be reconstituted in a 100 ml (for subjects with a body weight of 20 kg or more) or 50 ml (for subjects under 20 kg of body weight) infusion bag. It will be administered as an intravenous infusion using an infusion pump over a period of 50 minutes. In subjects with a BMI over 30 kg/m2, infusion will be performed over a period of 100 minutes

Intervention Type OTHER

Other Intervention Names

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Active Control

Eligibility Criteria

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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).
Minimum Eligible Age

9 Months

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Exeltis

INDUSTRY

Sponsor Role collaborator

Chemo Research

INDUSTRY

Sponsor Role collaborator

Linical

UNKNOWN

Sponsor Role collaborator

KLIXAR

UNKNOWN

Sponsor Role collaborator

Inmunova S.A.

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Santiago Sanguineti, Ph.D

Role: STUDY_DIRECTOR

Inmunova S.A.

Locations

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Hospital Interzonal Dr. José Penna

Bahía Blanca, Buenos Aires, Argentina

Site Status NOT_YET_RECRUITING

Hospital Penna

Bahía Blanca, Buenos Aires, Argentina

Site Status NOT_YET_RECRUITING

Hospital de niños Sor María Ludovica

La Plata, Buenos Aires, Argentina

Site Status RECRUITING

Clinica del niño y la madre

Mar del Plata, Buenos Aires, Argentina

Site Status RECRUITING

Hospital Interzonal Especializado Materno Infantil Don Victorio Tetamanti

Mar del Plata, Buenos Aires, Argentina

Site Status RECRUITING

Sanatorio de la Trinidad Ramos Mejia

Ramos Mejía, Buenos Aires, Argentina

Site Status NOT_YET_RECRUITING

Hospital El Cruce

San Juan Bautista, Buenos Aires, Argentina

Site Status NOT_YET_RECRUITING

Sanatorio Güemes

Buenos Aires, Buenos Aires F.D., Argentina

Site Status ACTIVE_NOT_RECRUITING

Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan"

Buenos Aires, Buenos Aires F.D., Argentina

Site Status RECRUITING

Hospital General de Niños Pedro de Elizalde

Buenos Aires, Buenos Aires F.D., Argentina

Site Status ACTIVE_NOT_RECRUITING

Sanatorio Anchorena

CABA, Buenos Aires F.D., Argentina

Site Status ACTIVE_NOT_RECRUITING

Hospital Privado Centro Médico de Córdoba

Córdoba, Córdoba Province, Argentina

Site Status RECRUITING

Hospital de Niños de la Santísima Trinidad

Córdoba, Córdoba Province, Argentina

Site Status RECRUITING

Hospital "San Antonio de Padua" Río Cuarto

Río Cuarto, Córdoba Province, Argentina

Site Status RECRUITING

Hospital Pediátrico Dr. Humberto Notti

Mendoza, Mendoza Province, Argentina

Site Status RECRUITING

Hospital Teodoro J. Schestakow

San Rafael, Mendoza Province, Argentina

Site Status RECRUITING

Clínica Pediátrica San Lucas

Neuquén, Neuquén Province, Argentina

Site Status SUSPENDED

Hospital Público Materno Infantil

Salta, Salta Province, Argentina

Site Status RECRUITING

Hospital Pediátrico San Luis

San Luis, San Luis Province, Argentina

Site Status RECRUITING

Hospital de Niños Zona Norte "Dr. Roberto M. Carra"

Rosario, Santa Fe Province, Argentina

Site Status RECRUITING

Sanatorio de Niños

Rosario, Santa Fe Province, Argentina

Site Status RECRUITING

Hospital De Clínicas Pte. Nicolás Avellaneda

San Miguel de Tucumán, Tucumán Province, Argentina

Site Status ACTIVE_NOT_RECRUITING

Clínica Zabala

Ciudad Autonoma de Buenos Aire, , Argentina

Site Status ACTIVE_NOT_RECRUITING

Hospital de Niños Dr. Ricardo Gutierrez

Ciudad Autonoma de Buenos Aire, , Argentina

Site Status RECRUITING

Sanatorio Allende

Córdoba, , Argentina

Site Status WITHDRAWN

Cliniques universitaires Saint-Luc

Brussels, , Belgium

Site Status ACTIVE_NOT_RECRUITING

CHU De Bordeaux

Bordeaux, , France

Site Status ACTIVE_NOT_RECRUITING

Hospices Civils De Lyon - Hopital Femme Mere Enfant

Lyon, , France

Site Status RECRUITING

Centre Hospitalier Universitaire De Montpellier

Montpellier, , France

Site Status ACTIVE_NOT_RECRUITING

CHU Nantes

Nantes, , France

Site Status NOT_YET_RECRUITING

Hospital Necker Enfants Malades

Paris, , France

Site Status ACTIVE_NOT_RECRUITING

Robert Debre University Hospital

Paris, , France

Site Status RECRUITING

Trousseau Hospital

Paris, , France

Site Status ACTIVE_NOT_RECRUITING

CHU Rouen

Rouen, , France

Site Status NOT_YET_RECRUITING

Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status NOT_YET_RECRUITING

University Hospital Cologne AöR

Cologne, , Germany

Site Status NOT_YET_RECRUITING

Universitaetsklinikum Heidelberg AöR

Heidelberg, , Germany

Site Status RECRUITING

Children's Health Ireland

Dublin, , Ireland

Site Status ACTIVE_NOT_RECRUITING

University Hospital Consorziale Policlinico

Bari, , Italy

Site Status RECRUITING

IRCCS Sant'Orsola

Bologna, , Italy

Site Status NOT_YET_RECRUITING

Istituto Gianina Gaslini

Genova, , Italy

Site Status RECRUITING

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status RECRUITING

Azienda Ospedale Università

Padua, , Italy

Site Status NOT_YET_RECRUITING

Spitalul Clinic de Urgenta pentru Copii Marie Sklodowska Curie

Bucharest, , Romania

Site Status NOT_YET_RECRUITING

Spitalul Clinic De Urgenta Pentru Copii Cluj-Napoca

Cluj-Napoca, , Romania

Site Status RECRUITING

Spitalul Clinic de Urgenta pentru Copii Louis Turcanu

Timișoara, , Romania

Site Status NOT_YET_RECRUITING

Hospital Universitario De Cruces

Barakaldo, , Spain

Site Status RECRUITING

Hospital Infantil Universitario Niño Jesus

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario 12 De Octubre

Madrid, , Spain

Site Status RECRUITING

Bristol Royal Hospital for Children

Bristol, , United Kingdom

Site Status NOT_YET_RECRUITING

Royal Hospital for Sick Children

Glasgow, , United Kingdom

Site Status RECRUITING

Great Ormon Street Hospital for Children

London, , United Kingdom

Site Status NOT_YET_RECRUITING

Countries

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Argentina Belgium France Germany Ireland Italy Romania Spain United Kingdom

Central Contacts

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Mariana Colonna, Bioch

Role: CONTACT

+541120331455 ext. 6184

Ana Perez

Role: CONTACT

Other Identifiers

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CT-INM004-04

Identifier Type: -

Identifier Source: org_study_id

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