MedPath

Bezlotoxumab (MK-6072) Versus Placebo in Children With Clostridium Difficile Infection (CDI) (MK-6072-001)

Phase 3
Completed
Conditions
Clostridium Difficile Infection
Interventions
Biological: Bezlotoxumab
Drug: Placebo
Drug: Antibacterial drug treatment (ABD)
Registration Number
NCT03182907
Lead Sponsor
Merck Sharp & Dohme LLC
Brief Summary

The primary objectives of this study are to evaluate the pharmacokinetics (PK), safety, and tolerability of bezlotoxumab (MK-6072) in children aged 1 to \<18 years of age with a confirmed diagnosis of Clostridium difficile infection (CDI) who are receiving antibacterial drug treatment. The primary hypothesis is that the area under the concentration-time curve from 0 to infinity (AUC0-inf) of bezlotoxumab after treatment of pediatric participants with bezlotoxumab is similar when compared to the AUC0-inf of bezlotoxumab after treatment of adults with bezlotoxumab.

Detailed Description

Historical adult pharmacokinetic data is from NCT01241552 and NCT01513239.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
148
Inclusion Criteria
  • At screening has suspected or confirmed Clostridium difficile infection (CDI), and is receiving or is planning to receive a 10- to 21-day course of antibacterial drug treatment for CDI
  • At study infusion has a diagnosis of CDI confirmed by a diagnostic assay which detects the presence of C. difficile toxin in stool, and is still receiving antibacterial drug treatment for CDI
  • Female is not pregnant, and not breastfeeding; but if of childbearing potential agrees to follow contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study treatment
  • Participant and/or parent or caregiver must be able to read, understand, and complete the daily diary
Exclusion Criteria
  • Has an uncontrolled chronic diarrheal illness
  • Has a known hypersensitivity to bezlotoxumab, its active substance and/or any of its excipients
  • At randomization, their planned course of antibacterial drug treatment for CDI is longer than 21 days
  • At screening has received any listed prohibited prior and concomitant treatments and procedures
  • Has previously participated in this study, has previously received bezlotoxumab, has received an experimental monoclonal antibody against C. difficile toxin B, or has received a vaccine directed against C. difficile or its toxins.
  • Has received an investigational study agent within the previous 30 days, or is currently participating in or scheduled to participate in any other clinical study with an investigational agent during the 12-week study period

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
BezlotoxumabBezlotoxumabParticipants receive 10 mg of bezlotoxumab per kg body weight via a single 60-minute (±10 minutes) intravenous (IV) infusion on Day 1. Additionally, participants receive background antibacterial drug treatment (ABD) for 10-21 days per institutional guidelines, at the investigator's discretion. Dose may then be changed based on results from initial 12 participants.
BezlotoxumabAntibacterial drug treatment (ABD)Participants receive 10 mg of bezlotoxumab per kg body weight via a single 60-minute (±10 minutes) intravenous (IV) infusion on Day 1. Additionally, participants receive background antibacterial drug treatment (ABD) for 10-21 days per institutional guidelines, at the investigator's discretion. Dose may then be changed based on results from initial 12 participants.
PlaceboPlaceboParticipants receive placebo for bezlotoxumab consisting of either 0.9% sodium chloride or 5% dextrose via a single 60-minute (±10 minutes) IV infusion on Day 1. Additionally, participants receive background ABD for 10-21 days per institutional guidelines, at the investigator's discretion.
PlaceboAntibacterial drug treatment (ABD)Participants receive placebo for bezlotoxumab consisting of either 0.9% sodium chloride or 5% dextrose via a single 60-minute (±10 minutes) IV infusion on Day 1. Additionally, participants receive background ABD for 10-21 days per institutional guidelines, at the investigator's discretion.
Primary Outcome Measures
NameTimeMethod
Area Under the Concentration-Time Curve of Bezlotoxumab From Time 0 to Infinity (AUC0-inf)Day 1 (2 hours postdose), Days 10, 29, 57, and 85

Blood samples were collected at specified intervals for the determination of AUC0-inf. AUC0-inf was defined as the area under the concentration-time curve of bezlotoxumab from time zero to infinity. Per protocol, AUC0-inf of bezlotoxumab was determined for each age cohort.

Percentage of Participants Who Experienced an Adverse Event (AE)Up to approximately 12 weeks

An AE was defined as any untoward medical occurrence associated with the use of a drug in a participant, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product and does not imply any judgment about causality. Per protocol, percentage of participants with AEs in the bezlotoxumab and placebo groups were presented.

Percentage of Participants Who Discontinued Study Due to an AEUp to approximately 12 weeks

An AE was defined as any untoward medical occurrence associated with the use of a drug in a participant, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product and does not imply any judgment about causality. Per protocol, percentage of participants who discontinued study due to AEs in the bezlotoxumab and placebo groups were presented.

Secondary Outcome Measures
NameTimeMethod
Percentage of High-Risk Participants Who Experienced a SCRUp to approximately 12 Weeks

SCR was ICR of baseline CDI episode (improvement in number and character of bowel movements and doesn't require further CDI therapy within 2 days after completion of up to 21 days of ABD for CDI) and no CDI recurrence (diarrhea recurrence with positive stool test for C. difficile toxin, for which participant, in investigator's opinion, requires and receives ABD for CDI). High-risk was meeting ≥1 criteria: a) was immunocompromised b) had ≥1 episodes of CDI prior to baseline episode c) had severe CDI baseline episode d) had C. difficile ribotype027 in stool at baseline CDI episode e) received ≥1 systemic ABD known to increase CDI risk. Per protocol, percentage of high-risk participants who had SCR in bezlotoxumab and placebo groups were presented.

Percentage of Participants Who Had Positive Antibodies to BezlotoxumabUp to approximately 12 Weeks

Blood samples were collected to determine antibodies to bezlotoxumab. Per protocol, percentage of participants with treatment-emergent positive antibodies to bezlotoxumab following single infusion of bezlotoxumab were reported for each age cohort.

Percentage of Participants Who Had a Clostridium Difficile Infection (CDI) RecurrenceUp to approximately 12 Weeks

CDI recurrence was defined as diarrhea recurrence (new episode of diarrhea after initial clinical response \[ICR\] as defined by a change in normal bowel habits for ≥2 days with either watery diarrhea or at least 6 unformed bowel movements \[UBMs\] within 48-hours) associated with a positive stool test for C. difficile toxin, and for which the participant, in the investigator's opinion, requires and receives ABD treatment for CDI. Per protocol, percentage of participants who had a CDI recurrence in the bezlotoxumab and placebo groups were reported.

Percentage of Participants Who Had a Sustained Clinical Response (SCR)Up to approximately 12 Weeks

SCR was defined as the ICR of baseline CDI episode (improvement in number and character of bowel movements and doesn't require further CDI therapy within 2 days after completion of up to 21 days of ABD treatment for CDI) and no CDI recurrence (diarrhea recurrence associated with a positive stool test for C. difficile toxin, and for which the participant, in investigator's opinion, requires and receives ABD for CDI). Per protocol, percentage of participants who had a SCR in bezlotoxumab and placebo groups were presented.

Percentage of High-Risk Participants Who Experienced a CDI RecurrenceUp to approximately 12 Weeks

CDI recurrence was defined as diarrhea recurrence (new diarrhea episode after ICR defined by change in normal bowel habits for ≥2 days with watery diarrhea or at least 6 UBMs within 48-hours) with positive stool test for C. difficile toxin for which participant, in investigator's opinion, requires and receives ABD for CDI. High-risk was meeting ≥1 criteria at/before randomization: a) was immunocompromised b) had ≥1 CDI episode prior to baseline episode c) had severe CDI baseline episode d) had C. difficile ribotype027 in stool at baseline CDI episode e) received ≥1 systemic ABD known to increase CDI risk. Per protocol, percentage of high-risk participants who experienced a CDI recurrence in the bezlotoxumab and placebo groups were presented.

Percentage of Participants Who Experienced One or More Infusion Related ReactionUp to approximately 24 hours after infusion on Day 1

Infusion related reaction included events meeting any of the 3 criteria: 1) acute onset of an illness involving skin, mucosal tissue, or both and at least 1 of the following: respiratory compromise, reduced blood pressure (BP) or associated symptoms of end-organ dysfunction 2) two or more of the following after onset of study infusion: involving skin-mucosal tissue, respiratory compromise, reduced BP or associated symptoms, or persistent gastrointestinal symptoms 3) reduced BP after onset of infusion or \>30% decrease in systolic BP from baseline. Per protocol, percentage of participants experiencing 1 or more infusion-related reactions within 24 hours following the start of study medication infusion were reported.

Trial Locations

Locations (75)

Red Cross War Memorial Children's Hospital ( Site 2601)

🇿🇦

Cape Town, Western Cape, South Africa

Hospital Privado de Cordoba ( Site 2102)

🇦🇷

Cordoba, Argentina

Hospital Pablo Tobon Uribe-Infectology pediatric ( Site 2166)

🇨🇴

Medellin, Antioquia, Colombia

Hospital Kuala Lumpur ( Site 3100)

🇲🇾

Kuala Lumpur, Malaysia

Hospital de Braga ( Site 1600)

🇵🇹

Braga, Portugal

Tufts Medical Center-Floating Hospital for Children ( Site 0046)

🇺🇸

Boston, Massachusetts, United States

University Hospitals Cleveland Medical Center ( Site 0029)

🇺🇸

Cleveland, Ohio, United States

Vanderbilt University Medical Center ( Site 0022)

🇺🇸

Nashville, Tennessee, United States

The Children's Hospital of San Antonio ( Site 0009)

🇺🇸

San Antonio, Texas, United States

Primary Children's Hospital ( Site 0001)

🇺🇸

Salt Lake City, Utah, United States

Hospital Universitario da Universidade Federal de Santa Maria ( Site 0209)

🇧🇷

Santa Maria, Rio Grande Do Sul, Brazil

Santa Casa de Misericordia de Belo Horizonte ( Site 0208)

🇧🇷

Belo Horizonte, Minas Gerais, Brazil

Children's Center for Digestive Healthcare ( Site 0052)

🇺🇸

Atlanta, Georgia, United States

UCSF Medical Center ( Site 0049)

🇺🇸

San Francisco, California, United States

Our Lady of the Lake Hospital ( Site 0007)

🇺🇸

Baton Rouge, Louisiana, United States

University of Chicago ( Site 0019)

🇺🇸

Chicago, Illinois, United States

Montefiore Einstein Center ( Site 0041)

🇺🇸

Bronx, New York, United States

Seattle Childrens Hospital ( Site 0028)

🇺🇸

Seattle, Washington, United States

Fakultni Nemocnice Brno Bohunice ( Site 2000)

🇨🇿

Brno, Brno-mesto, Czechia

Hospital de Clinicas da Universidade Federal do Parana ( Site 0203)

🇧🇷

Curitiba, Parana, Brazil

Instituto de Oncologia Pediatrica - GRAACC - Unifesp ( Site 0205)

🇧🇷

Sao Paulo, Brazil

Universitaetsklinikum Muenster ( Site 1400)

🇩🇪

Muenster, Nordrhein-Westfalen, Germany

Universitaetsklinikum Hamburg Eppendorf ( Site 1402)

🇩🇪

Hamburg, Germany

Del-pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet ( Site 2202)

🇭🇺

Budapest, Hungary

Hospital Universitario "Dr. Jose Eleuterio Gonzalez" ( Site 0508)

🇲🇽

Monterrey, Nuevo Leon, Mexico

SPZOZ im. Dzieci Warszawy w Dziekanowie Lesnym ( Site 2405)

🇵🇱

Lomianki, Mazowieckie, Poland

Fakultni nemocnice Plzen ( Site 2001)

🇨🇿

Plzen Lochotin, Plzensky Kraj, Czechia

Children's Hospital - Colorado ( Site 0013)

🇺🇸

Aurora, Colorado, United States

Mayo Clinic - Rochester ( Site 0004)

🇺🇸

Rochester, Minnesota, United States

Washington University ( Site 0037)

🇺🇸

Saint Louis, Missouri, United States

Children's Hospital - Los Angeles ( Site 0021)

🇺🇸

Los Angeles, California, United States

The Johns Hopkins Rubenstein Child Health Building ( Site 0034)

🇺🇸

Baltimore, Maryland, United States

Columbia University Medical Center/ MSCHONY ( Site 0042)

🇺🇸

New York, New York, United States

SUNY Upstate Medical Center, University Hospital ( Site 0027)

🇺🇸

Syracuse, New York, United States

Cincinnati Children's Hospital Medical Center ( Site 0024)

🇺🇸

Cincinnati, Ohio, United States

Duke University Health System ( Site 0025)

🇺🇸

Durham, North Carolina, United States

St. Jude Children's Research Hospital ( Site 0050)

🇺🇸

Memphis, Tennessee, United States

Fundacion Valle del Lili ( Site 2161)

🇨🇴

Cali, Valle Del Cauca, Colombia

Hospital Italiano de Buenos Aires. ( Site 2103)

🇦🇷

Caba, Buenos Aires, Argentina

Fundacion Santa Fe de Bogota ( Site 2167)

🇨🇴

Bogotá, Distrito Capital De Bogota, Colombia

Hospital Pequeno Principe ( Site 0200)

🇧🇷

Curitiba, Parana, Brazil

Sabah Womens & Childrens Hospital ( Site 3101)

🇲🇾

Kota Kinabalu, Sabah, Malaysia

Fundacion Cardioinfantil Instituto de Cardiologia ( Site 2163)

🇨🇴

Bogota, Distrito Capital De Bogota, Colombia

2. LF UK a FN Motol ( Site 2003)

🇨🇿

Praha 5, Czechia

Instituto Nacional de Pediatria ( Site 0503)

🇲🇽

Mexico City, Mexico

Oslo universitetssykehus ( Site 1500)

🇳🇴

Oslo, Norway

Instytut Pomnik Centrum Zdrowia Dziecka ( Site 2406)

🇵🇱

Warszawa, Mazowieckie, Poland

Wojewodzki Specjalistyczny Szpital Dzieciecy ( Site 2410)

🇵🇱

Olsztyn, Warminsko-mazurskie, Poland

Centro Hospitalar de Lisboa Central EPE. Hospital D. Estefania ( Site 1601)

🇵🇹

Lisboa, Portugal

Inst. Portugues de Oncologia de Lisboa Francisco Gentil EPE ( Site 1605)

🇵🇹

Lisboa, Portugal

Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E. ( Site 1603)

🇵🇹

Porto, Portugal

Spitalul Clinic de Boli Infectioase Cluj-Napoca ( Site 2502)

🇷🇴

Cluj-Napoca, Cluj, Romania

Spitalul Clinic de Boli Infectioase si Tropicale Dr. Victor Babes ( Site 2500)

🇷🇴

Bucuresti, Romania

Phoenix Pharma Pty Ltd ( Site 2607)

🇿🇦

Port Elizabeth, Eastern Cape, South Africa

Johese Clinical Research ( Site 2605)

🇿🇦

Centurion, Gauteng, South Africa

Institutul National de Boli Infectioase Prof. Dr. Matei Bals ( Site 2501)

🇷🇴

Bucuresti, Romania

Molotlegi Street ( Site 2603)

🇿🇦

Pretoria, Gauteng, South Africa

Chris Hani Baragwanath Academic Hospital ( Site 2602)

🇿🇦

Johannesburg, Gauteng, South Africa

Hospital Universitario Sant Joan de Deu ( Site 1704)

🇪🇸

Esplugues de Llobregat, Barcelona, Spain

Hospital Universitario La Paz ( Site 1703)

🇪🇸

Madrid, Spain

ITCC Barnokologen Astrid Lindgrens Barnsjukhus NKS ( Site 1800)

🇸🇪

Stockholm, Stockholms Lan, Sweden

Hospital Infantil Universitario Nino Jesus ( Site 1701)

🇪🇸

Madrid, Spain

Hospital Universitario Virgen del Rocio ( Site 1705)

🇪🇸

Sevilla, Spain

Barncancercentrum ( Site 1801)

🇸🇪

Goteborg, Vastra Gotalands Lan, Sweden

Southampton General Hospital ( Site 1900)

🇬🇧

Southampton, Worcestershire, United Kingdom

Leeds Teaching Hospitals NHS Trust ( Site 1901)

🇬🇧

Leeds, United Kingdom

SZTE Szent-Gyorgyi Albert Klinikai Kozpont ( Site 2200)

🇭🇺

Szeged, Csongrad, Hungary

Instituto Tecnologico y de Estudios Superiores de Monterrey ( Site 0502)

🇲🇽

Monterrey, Nuevo Leon, Mexico

Hospital Infantil de Mexico Federico Gomez ( Site 0501)

🇲🇽

Mexico City, Mexico

Haukeland universitetssykehus ( Site 1501)

🇳🇴

Bergen, Vestfold, Norway

Wojewodzki Szpital Obserwacyjno Zakazny ( Site 2404)

🇵🇱

Bydgoszcz, Kujawsko-pomorskie, Poland

Wojewodzki Specjalistyczny Szpital im. dr W. Bieganskiego w Lodzi ( Site 2400)

🇵🇱

Lodz, Lodzkie, Poland

Spitalul Clinic de Boli Infectioase Constanta ( Site 2504)

🇷🇴

Constanta, Romania

Centro Medico Imbanaco ( Site 2160)

🇨🇴

Cali, Valle Del Cauca, Colombia

Semmelweis University-II.sz. Gyermekgyógyászati Klinika ( Site 2201)

🇭🇺

Budapest, Hungary

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