Study of Chimeric Monoclonal Antibodies to Shiga Toxins 1 and 2

Thallion Pharmaceuticals45 enrolled

Overview

This study is designed to evaluate the safety and efficacy of cαStx1 and cαStx2 administered concomitantly in children presenting early signs of Shiga Toxin-Producing Bacterial (STPB) Infection.

Currently, there is no etiological treatment of STPB-induced HUS. Ideally, such treatment would be started in the early phase of the infection and would protect against both types of toxins and all of their variants. The chimeric anti-Shiga toxins 1 (cαStx1) and 2 (cαStx2) antibodies are intended to be administered as a single infusion and provide simultaneous protection against the two Shiga toxins (Stx1 and Stx2) by decreasing the incidence and severity of Shiga toxin-mediated clinical events including bloody diarrhea/hemorrhagic colitis and Hemolytic Uremic Syndrome (HUS) and associated sequelae.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Shiga Toxin Producing Bacterial Infection

Interventions

cαStx1/cαStx2DRUG

cαStx1/cαStx2 administered concomitantly at a dose of 1 mg/kg (low dose cohort) or 3 mg/kg (high dose cohort)per antibody over 1 hour + standard of care

PlaceboDRUG

Placebo administered over 1 hour + standard of care

Eligibility

Sex: ALLMin age: 6 MonthsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Bloody diarrhea (by visual inspection) for no more than 36 hours prior to screening (signature of the informed consent). 2. Detection of Shiga toxin (Stx1 and/or Stx2) in stool Exclusion Criteria: 1. Laboratory findings compatible with development of at least two out of three following criteria that define Hemolytic Uremic Syndrome (HUS): Hemolytic Anemia: hematocrit \< 30% with evidence of hemolysis (as indicated by Lactate Dehydrogenase (LDH) above the upper limit of normal for age or the finding of schistocytes on peripheral smear); Thrombocytopenia: platelet count \<150 x 103/uL; Nephropathy: serum creatinine \> Upper Limit Normal (ULN) adjusted for age and gender. 2. Bloody-diarrhea suspected not to be caused by Shiga Toxin-Producing Bacteria (STPB) but by other organisms or preexisting diseases. 3. Family history of proven or suspected hereditary Hemolytic Uremic Syndrome (HUS) or thrombotic thrombocytopenic purpura (TTP). 4. History of chronic/recurrent hemolytic anemia or thrombocytopenia.

Locations (11)

Unnamed facility

Bahía Blanca, Argentina

Unnamed facility

Buenos Aires, Argentina

Unnamed facility

Córdoba, Argentina

Unnamed facility

La Plata, Argentina

Outcomes

Primary Outcomes

Safety and Tolerability: Evaluation of number and type of adverse events and serious adverse events between arms and dosage cohorts

Evaluation of the safety and tolerability of two different intravenous dose levels of a combined cαStx1/cαStx2 preparation in separate groups of children presenting with Shiga Toxin-Producing Bacterial (STPB) infection.

Time frame: Up to 1 year

Secondary Outcomes

Efficacy: Comparison of clinical event rates (Hemolytic Uremic Syndrome, Bloody Diarrhea) and associated sequelae between arms and dosage cohorts in children presenting with Shiga Toxin-Producing Bacterial (STPB) infection.

Time frame: Up to 1 year

Data from ClinicalTrials.gov

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