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    Summary
    EudraCT Number:2022-001504-17
    Sponsor's Protocol Code Number:16027
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-04-08
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2022-001504-17
    A.3Full title of the trial
    Prospective, historically controlled study to evaluate the efficacy and safety of a new pediatric formulation of nifurtimox in children aged 0 to 17 years with Chagas’ disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to learn how well a new formulation of drug nifurtimox for children works and how safe it is in children aged 0 to 17 years with Chagas’ disease
    A.4.1Sponsor's protocol code number16027
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02625974
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/081/2022
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBayer AG
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBayer AG
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBayer AG
    B.5.2Functional name of contact pointBayer Clinical Trials Contact
    B.5.3 Address:
    B.5.3.1Street AddressN/A
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code13342
    B.5.3.4CountryGermany
    B.5.4Telephone number+4930300139003
    B.5.6E-mailclinical-trials-contact@bayer.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Lampit®
    D.2.1.1.2Name of the Marketing Authorisation holderBayer AG
    D.2.1.2Country which granted the Marketing AuthorisationArgentina
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNifurtimox
    D.3.2Product code BAY A2502
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNifurtimox
    D.3.9.1CAS number 23256-30-6
    D.3.9.2Current sponsor codeBAY A2502
    D.3.9.4EV Substance CodeSUB09280MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNifurtimox
    D.3.9.1CAS number 23256-30-6
    D.3.9.2Current sponsor codeBAY A2502
    D.3.9.4EV Substance CodeSUB09280MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chagas disease
    E.1.1.1Medical condition in easily understood language
    An inflammatory, infectious disease caused by the parasite Trypanosoma cruzi
    E.1.1.2Therapeutic area Diseases [C] - Parasitic Diseases [C03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10008384
    E.1.2Term Chagas' disease
    E.1.2System Organ Class 100000004862
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the superiority of a 60-day regimen of nifurtimox to historical untreated control at the 12-month follow-up (360 days from end of treatment [EOT]) as seroreduction (defined as a ≥ 20% reduction in optical density [OD] measured by conventional enzyme-linked immune sorbent assay [ELISA]) compared to baseline in subjects ≥8 months to <18 years of age at randomization; or sero-conversion (defined as negative Immunoglobulin G [IgG] concentration) in all subjects
    E.2.2Secondary objectives of the trial
    - To assess the comparability of a 30-day regimen of nifurtimox to a 60-day regimen of nifurtimox as sero-reduction or sero-conversion at the 12-month follow-up (360 days from EOT)
    - To evaluate the safety/tolerability profile of nifurtimox by laboratory parameters (hematology, blood chemistry, urinalysis), electrocardiogram (ECG) monitoring, vital sign measurements (blood pressure, heart rate, respiratory rate, temperature), adverse
    event (AE) monitoring, and physical examinations, including neurological examinations
    - To evaluate the pharmacokinetics (PK)/pharmacodynamics (PD) of nifurtimox in children receiving the drug for treatment of Chagas’ disease (to be described in a separate report)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Part 1:
    - Male and female pediatric subjects aged 0 days to younger than 18 years
    - Chagas’ disease diagnosed/ confirmed by a) Subjects < 8 months of age at randomization must demonstrate direct observation of Trypanosoma cruzi by concentration test; b) Subjects ≥ 8 months to < 18 years of age at randomization must demonstrate a positive conventional ELISA result for both recombinant ELISA and total purified antigen ELISA
    Part 2:
    - Male and female subjects who were randomized and received at least one dose of their assigned 60- or 30-day regimen of nifurtimox treatment
    E.4Principal exclusion criteria
    Part 1:
    - Subjects aged 0 to 27 days who, at birth, were pre-term, weighed less than 2500 g, or had a maximum Apgar score < 7 at 5 minutes
    - Subjects with any of the following conditions that is associated with Chagas’ disease, such as: Known evidence of Chagas’ disease-related cardiomyopathy/ Chagas’ heart disease; Known evidence of Chagas’ disease-related gastrointestinal dysfunction (e.g. megaoesophagus, megacolon, or both) or Chagas’ digestive disease; Serious manifestations of acute Chagas’ disease, including myocarditis, meningoencephalitis, or pneumonitis; Known evidence of Chagas’ disease-related damage to the peripheral nervous system or peripheral neuropathy; Clinically significant psychiatric disorder (e.g. moderate to severe depression, severe anxiety, or psychosis) or epilepsy
    - Subjects with contraindications/ warnings to nifurtimox administration, or with conditions that may increase the risk of the undesirable effects of nifurtimox
    - Subjects who have had previous treatment with trypanocidal agents or an accepted indication for antiparasitic therapy (e.g. reactivation of Chagas' infection due to immunosuppression by several diseases or treatment with steroids)
    - Subjects living in housing conditions where there is no active or effective vector-control to Trypanosoma cruzi reinfection as determined by Ministry of Health guidelines in each country
    Part 2:
    - Subjects with acute or chronic health conditions or congenital disorders which, in the opinion of the investigator, would make them unsuitable for participation in the clinical study
    - Subjects living in housing conditions where there is no active or effective vector-control to Trypanosoma cruzi reinfection as determined by Ministry of Health guideline of the respective country
    - Subjects with clinical manifestations of Chagas’ disease, such as: Known evidence of Chagas’ disease-related gastrointestinal dysfunction; Serious manifestations of acute Chagas’ disease, including myocarditis, meningoencephalitis, or pneumonitis
    - Immuno-compromised subjects (e.g. those with human immunodeficiency virus infection primary immunodeficiency, or prolonged treatment with corticosteroids or other immunosuppressive drugs)
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage of subjects with sero-reduction or sero-conversion
    - Incidence rate of seronegative conversion for subjects received at least one dose of the 60-day nifurtimox treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    - 12 months post-treatment
    - 48 months post-treatment
    E.5.2Secondary end point(s)
    Part 1:
    - Clinical signs/symptoms of Chagas’ disease
    - Concentration test for Trypanosoma cruzi (subjects < 8 months of age at randomization)
    - Serological response using non-conventional enzyme-linked immunosorbent assay-F29 (ELISAF29) test
    - Disease state determined by qPCR
    - Treatment emergent adverse events and treatment-emergent serious adverse events
    - Hematology, blood chemistry, coagulation and urinalysis abnormalities
    - ECG abnormalities
    - Vital signs
    - Plasma nifurtimox concentrations
    Part 2:
    - Incidence rate of seronegative conversion in subjects who received at least one dose of the 30-day nifurtimox treatment regimen
    - Proportion of responders who show both seronegative conversion and no evidence of established cardiomyopathy
    - Serial reduction of optical density
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1:
    - Up to Visit 11 post-treatment
    - Up to Visit 9 post-treatment
    - Up to Visit 11 post-treatment
    - Up to Visit 11 post-treatment
    - Up to Visit 11 post-treatment
    - Up to Visit 11 post-treatment
    - Up to Visit 11 post-treatment
    - Up to Visit 11 post-treatment
    - Pre-dose and up to 8 hours post-dose
    Part 2:
    - 48 months post-treatment
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    historical placebo control
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Argentina
    Bolivia, Plurinational State of
    Colombia
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days15
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 330
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 7
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 37
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 143
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 143
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under age incapable of giving consent personally.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 330
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Colombia
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