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Effects of JNJ-53718678 in Adult and Adolescent Participants Who Had a Hematopoietic Stem Cell Transplantation and Who Are Infected With Respiratory Syncytial Virus (RSV)

Phase 2
Terminated
Conditions
Respiratory Syncytial Virus Infections
Interventions
Drug: JNJ-53718678 250 mg
Drug: Placebo
Drug: JNJ-53718678 125 mg
Registration Number
NCT04056611
Lead Sponsor
Janssen Sciences Ireland UC
Brief Summary

The purpose of this study is to evaluate the effect of JNJ-53718678 on the development of respiratory syncytial virus (RSV) lower respiratory tract infection (LRTIs) in adult hematopoietic stem cell transplant (HSCT) recipients with RSV upper RTI.

Detailed Description

RSV is recognized as major respiratory pathogen in infants and young children and causes upper and lower respiratory illness among all age groups, often going undiagnosed. Immunocompromised (IC) participants have a reduced ability to combat infection due to an impaired or weakened immune system. Within the IC population, HSCT recipients are generally regarded as having a particularly high risk for more severe disease caused by RSV, representing a substantial unmet need for antiviral treatment of RSV infections in this participant population. JNJ-53718678 is an investigational, potent, small molecule, respiratory syncytial virus (RSV)-specific fusion inhibitor. The study will include a Screening Period (Day -2 to Day 1), a Treatment Period (Day 1 to Day 21), and a Follow-up Period (1 year). Assessments like chest X-ray, pulse/heart rate, respiratory rate, electrocardiogram (ECG), etc will be performed. Safety and efficacy will be assessed through the study. The total study duration for each participant will be approximately 49 days.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
3
Inclusion Criteria
  • Received an autologous or allogeneic hematopoietic stem cell transplant (HSCT) using any conditioning regimen
  • Absolute lymphocyte count (ALC) less than (<) 1,000 cells/microliter (mL)
  • Participant has laboratory confirmed RSV diagnosis within 48 hours of randomization
  • New onset of at least 1 of the following respiratory symptoms within 4 days prior to the anticipated start of dosing nasal congestion, rhinorrhea, cough or pharyngitis (sore throat), and/or worsening of one of these chronic (associated with previously existing diagnosis, example, chronic rhinorrhea, seasonal allergies, chronic lung disease) respiratory symptoms within 4 days prior to the anticipated start of dosing
  • Peripheral capillary oxygen saturation (SpO2) greater than or equal to (>=) 92 percent (%) on room air
Exclusion Criteria
  • Admitted to the hospital primarily for a lower respiratory tract disease of any cause as determined by the investigator
  • Requires supplemental oxygen at Screening or any time between Screening and randomization
  • Documented to be positive for other respiratory viruses (limited to influenza, parainfluenza, human rhinovirus, adenovirus, human metapneumovirus, or coronavirus) within 7 days prior to or at the Screening visit, if determined by local SOC testing (additional testing is not required)
  • Clinically significant bacteremia or fungemia within 7 days prior to or at Screening that has not been adequately treated, as determined by the investigator

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Adult cohort: JNJ-53718678 or PlaceboJNJ-53718678 250 mgParticipants greater than or equal to (\>=) 18 to less than or equal to (\<=) 75 years of age will receive 250 milligram (mg) JNJ-53718678 twice daily (bid) for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg once daily (qd) for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Adult cohort: JNJ-53718678 or PlaceboPlaceboParticipants greater than or equal to (\>=) 18 to less than or equal to (\<=) 75 years of age will receive 250 milligram (mg) JNJ-53718678 twice daily (bid) for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg once daily (qd) for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Adult cohort: JNJ-53718678 or PlaceboJNJ-53718678 125 mgParticipants greater than or equal to (\>=) 18 to less than or equal to (\<=) 75 years of age will receive 250 milligram (mg) JNJ-53718678 twice daily (bid) for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg once daily (qd) for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Adolescent cohort: JNJ-53718678 or PlaceboPlaceboParticipants \>=13 to \<18 years of age will receive 250 mg JNJ-53718678 bid for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg qd for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Adolescent cohort: JNJ-53718678 or PlaceboJNJ-53718678 250 mgParticipants \>=13 to \<18 years of age will receive 250 mg JNJ-53718678 bid for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg qd for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Adolescent cohort: JNJ-53718678 or PlaceboJNJ-53718678 125 mgParticipants \>=13 to \<18 years of age will receive 250 mg JNJ-53718678 bid for 21 days (without coadministration with moderate or strong CYP3A4 inhibitors), or 125 mg JNJ-53718678 bid for 21 days (coadministered with moderate or strong CYP3A4 inhibitors with the exception of posaconazole), or 125 mg qd for 21 days (when coadministered with posaconazole), or matching placebo for 21 days.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Who Developed Respiratory Syncytial Virus (RSV) Lower Respiratory Tract Infection (LRTI)Up to Day 28

Percentage of participants who developed RSV LRTI was assessed. RSV LRTI was defined as the development of a lower respiratory sign or symptom (including decrease in oxygen saturation or increase in supplemental oxygen to maintain oxygen saturation, wheezing, rhonchi, rales, dyspnea, tachypnea, worsening cough) and positive RSV test from lower respiratory tract sample (example \[eg\], sputum, induced sputum, bronchoalveolar lavage (BAL), lung biopsy, or autopsy specimen) within +-4 days of a new chest image finding, compared to baseline, consistent with a LRTI; OR positive RSV test from lower respiratory tract sample (eg, sputum, induced sputum, BAL, lung biopsy, or autopsy specimen) only; OR positive RSV test from upper respiratory tract sample within ±4 days of a new chest image finding, compared to baseline, consistent with a RSV LRTI as determined by the Endpoint Adjudication Committee (EAC).

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants Who Developed RSV-associated Lower Respiratory Tract Complication (LRTC)Up to Day 28

Percentage of participants who developed RSV-associated LRTC was assessed. RSV-associated LRTC defined as development of lower respiratory sign/symptom (includes decrease in oxygen saturation/increase in supplemental oxygen to maintain oxygen saturation, wheezing, rhonchi, rales, dyspnea, tachypnea, and worsening cough) and met 1 of following subcategories determined by EAC: a) RSV LRTI, b) secondary bacterial LRTI (positive specimen for clinically significant bacterium within 4 days of new chest image finding, compared to baseline, consistent with LRTI), c) secondary LRTI due to unusual pathogens (positive specimen for clinically significant unusual organism within 4 days of new chest image finding, compared to baseline, consistent with LRTI), d) secondary LRTC of unknown etiology (new chest image finding than baseline, consistent with LRTI, inflammatory process/ some other clinically significant pulmonary process which were absent within 4 days of new chest image finding).

Number of Participants With Treatment-emergent Adverse Events (TEAEs)Up to Day 49

An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the intervention. Any AE which occurred post first dose administration of study drug up to the end of study (EOS) (that is, Day 49) was considered as treatment-emergent.

Percentage of Participants With Treatment-emergent Abnormal (>=Grade 3) Clinical Laboratory FindingsUp to Day 49

Percentage of participants with greater than or equal to (\>=) Grade 3 treatment-emergent laboratory abnormalities (platelet count decreased, glucose increase) was assessed in this outcome measure. Treatment-emergent: any abnormality occurred post first dose of study drug up to end of study (that is, Day 49).

Percentage of Participants With Clinically Significant Abnormalities in Electrocardiogram (ECG) FindingsUp to Day 49

Percentage of participants with clinically significant abnormalities in ECG findings was assessed in this outcome measure. Various ECG variables assessed were heart rate: abnormally low (\<= 45 beats per minute \[bpm\]), abnormally high (\>= 120 bpm); PR interval: abnormally high (\>=210 milliseconds \[msec\]); QRS interval: abnormally high (\>=120 msec), QT interval and corrected QT (QTcF; according to Fridericia's formula) interval (\>450 msec, \>480 msec, or \>500 msec, increases from baseline \>30 msec or \>60 msec.)

Percentage of Participants Who Progressed to Respiratory Failure (of Any Cause) Requiring Mechanical Ventilation (Invasive or Noninvasive) and/or Death Among Those Who Developed RSV LRTI or RSV-associated LRTC Per the EAC's AssessmentUp to Day 49

Percentage of participants who progressed to respiratory failure (of any cause) requiring mechanical ventilation (invasive or noninvasive) and/or death among those who developed RSV LRTI or RSV-associated LRTC per the EAC's assessment was assessed. Here, '0' in the 'number of participants analyzed' field (N=0) signifies that no participants were available for the analysis because none of the participants developed RSV LRTI or RSV-associated LRTC per the EAC's Assessment.

Percentage of Participants With Treatment-emergent Abnormal Vital Signs FindingsUp to Day 49

Percentage of participants with abnormal vital signs findings was assessed. Abnormal vital parameters included pulse rate: abnormally low \<=45 bpm, abnormally high \>=120 bpm; Systolic Blood Pressure (SBP): abnormally low \<=90 Millimeter of mercury (mmHg), Grade 1 (mild): \> 90 mmHg - \< 100 mmHg, Grade 2 (moderate): \>= 100 mmHg to \<110 mmHg, Grade 3 (severe): \>=110 mmHg; Diastolic BP: abnormally low \<=50 mmHg, Grade 1: \>90 mmHg to \<100 mmHg, Grade 2: \>=100 mmHg to \<110 mmHg, Grade 3: \>=110 mmHg; Respiratory rate- Grade 1 (mild): 17-20 breaths per minute, Grade 2 (moderate): 21-25 breaths per minute, Grade 3 (severe): \>25 breaths per minute, Grade 4 (potentially life threatening): intubation; Temperature: abnormally high \>38.0 degree celsius. Vital signs abnormalities reported for at least 1 participant were reported in this outcome measure. Treatment-emergent: any abnormality occurred post first dose of study drug up to EOS (that is, Day 49).

Percentage of Participants Who Progressed to Respiratory Failure (of Any Cause) Requiring Mechanical Ventilation (Invasive or Noninvasive) and/or Death (All-cause Mortality)Up to Day 49

Percentage of participants who progressed to respiratory failure (of any cause) requiring mechanical ventilation (invasive or noninvasive) and/or death (all-cause mortality) was assessed.

Percentage of Participants Who Progressed to Death (All-cause Mortality) Among Those Who Developed RSV LRTI or RSV-associated LRTC Per the EAC's AssessmentUp to Day 49

Percentage of participants who progressed to death (all-cause mortality) among those who developed RSV LRTI or RSV-associated LRTC per the EAC's assessment was assessed. Here, '0' in the 'number of participants analyzed' field (N=0) signifies that no participants were available for the analysis because none of the participants developed RSV LRTI or RSV-associated LRTC per the EAC's Assessment.

Percentage of Participants Who Progressed to Death (All-cause Mortality)Up to Day 49

Percentage of participants who progressed to death (all-cause mortality) was assessed.

Percentage of Participants Who Progressed to Respiratory Failure (of Any Cause) Requiring Mechanical Ventilation (Invasive or Noninvasive) Among Those Who Developed RSV LRTI or RSV-associated LRTC Per the EAC's AssessmentUp to Day 49

Percentage of participants who progressed to respiratory failure (of any cause) requiring mechanical ventilation (invasive or noninvasive) among those who developed RSV LRTI or RSV-associated LRTC per the EAC's assessment was assessed. Here, '0' in the 'number of participants analyzed' field (N=0) signifies that no participants were available for the analysis because none of the participants developed RSV LRTI or RSV-associated LRTC per the EAC's Assessment.

Percentage of Participants Who Progressed to Respiratory Failure (of Any Cause) Requiring Mechanical Ventilation (Invasive or Noninvasive)Up to Day 49

Percentage of participants who progressed to respiratory failure (of any cause) requiring mechanical ventilation (invasive or noninvasive) was assessed.

Number of Supplemental Oxygen Free DaysThrough Day 28

Number of supplemental oxygen free days was assessed. The number of supplemental oxygen free days was the number of days the participants did not receive/require supplemental oxygen during the first 28 days post treatment.

Percentage of Participants With Treatment-emergent Oxygen SupplementationUp to Day 49

Percentage of participants who required treatment-emergent oxygen supplementation (e.g., supplemental oxygen, noninvasive pressure ventilation, invasive mechanical ventilation \[tracheal tube, laryngeal mask or tracheostomy\]). Any AE which occurred post first dose administration of study drug up to the end of study (that is, Day 49) were considered as treatment-emergent.

Respiratory Rate Over TimeBaseline (Day 1), Days 15, 28, and 35

Respiratory rate over time was reported by investigator. In this outcome measure, only those timepoints in which individual participant had data were reported.

Heart Rate Over TimeBaseline (Day 1), Days 15, 28, and 35

Heart rate over time was reported by investigator. In this outcome measure, only those timepoints in which individual participant had data were reported.

Peripheral Capillary Oxygen Saturation (SpO2) Over TimeBaseline (Day 1), Days 15, 28, and 35

Peripheral capillary oxygen saturation (SpO2) over time was reported by investigator. In this outcome measure, only those timepoints in which individual participant had data were reported.

Body Temperature Over TimeBaseline (Day 1), Days 15, 28, and 35

Body temperature (in Degrees Celsius) over time was reported. In this outcome measure, only those timepoints in which individual participant had data were reported.

Percentage of Participants Hospitalized (of Participants Who Were Not Hospitalized at Baseline)Up to Day 49

Percentage of participants who were not hospitalized at baseline and required hospitalization during the study was assessed.

Percentage of Participants Who Were Re-hospitalizedUp to Day 49

Percentage of participants who were re-hospitalized (of participants who were hospitalized at baseline and discharged during the study and of participants who were not hospitalized at baseline and required hospitalization and were discharged during the study) was assessed in this outcome measure.

Duration of Hospital StayUp to Day 49

Duration (in days) of hospital stay was assessed.

Duration of Intensive Care Unit (ICU) StayUp to Day 49

Duration of ICU stay was assessed. Duration (in hours) was defined as total number of hours a participant was in ICU from first dose of study drug until study termination.

Number of Participants With Grade 3 and Grade 4 Treatment-emergent Adverse Events (TEAEs) in the Infections and Infestations System Organ ClassUp to Day 49

An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non investigational) product. An AE did not necessarily had a causal relationship with the intervention. Participants with Grade 3 or Grade 4 TEAE were assessed in this outcome measure. Any AE which occurred post first dose administration of study drug up to the end of study (that is, Day 49) were considered as treatment-emergent.

Number of Participants With Respiratory Related AEsUp to Day 49

Number of participants with respiratory related AEs (respiratory infections) was assessed.

Number of Participants With Thoracic-related AEsUp to Day 49

Number of participants with thoracic-related AEs was assessed.

Number of Participants With Antibiotic Use Among Those Who Developed RSV LRTI or RSV-Associated LRTC Per the EAC's AssessmentUp to Day 49

Number of participants with antibiotic use among those who developed RSV LRTI or RSV-associated LRTC per the EAC's assessment was assessed. Here, '0' in the 'number of participants analyzed' field (N=0) signifies that no participants were available for the analysis because none of the participants developed RSV LRTI or RSV-associated LRTC per the EAC's Assessment.

Plasma Concentration of JNJ-53718678Days 1, 3, 8, 15 and 22

Plasma Concentration of JNJ-53718678 was reported. In this outcome measure, only those timepoints in which individual participant had data were reported.

RSV Viral Load Over TimeBaseline (Day 1), Days 15, 28, and 35

RSV viral load (RSV B) was measured over time by quantitative reverse transcription polymerase chain reaction (qRT-PCR) in the nasal swab specimens collected at the clinic visits and at home. In this outcome measure, only those timepoints in which individual participant had data were reported.

Trial Locations

Locations (69)

Hosp. de La Santa Creu I Sant Pau

🇪🇸

Barcelona, Spain

Hosp. Gral. Univ. Gregorio Maranon

🇪🇸

Madrid, Spain

Hosp. Univ. 12 de Octubre

🇪🇸

Madrid, Spain

Hosp Clinico Univ de Salamanca

🇪🇸

Salamanca, Spain

Hosp. Univ. Marques de Valdecilla

🇪🇸

Santander, Spain

Hosp. Virgen Del Rocio

🇪🇸

Sevilla, Spain

Skanes universitetssjukhus

🇸🇪

Malmö, Sweden

Kaohsiung Medical University Hospital

🇨🇳

Kaohsiung, Taiwan

Ronald Reagan UCLA Medical Center

🇺🇸

Los Angeles, California, United States

Hospital das Clinicas de Porto Alegre

🇧🇷

Porto Alegre, Brazil

China Medical University Hospital

🇨🇳

Taichung, Taiwan

National Cheng Kung University Hospital

🇨🇳

Tainan, Taiwan

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

Kings College Hospital NHS Trust

🇬🇧

London, United Kingdom

Henry Ford Hospital - Hematology/oncology

🇺🇸

Detroit, Michigan, United States

Northwell Health Cancer Institute

🇺🇸

Lake Success, New York, United States

MD Anderson Cancer Center - University of Texas

🇺🇸

Houston, Texas, United States

Hospital Espanol De Bahia Blanca

🇦🇷

Bahia Blanca, Argentina

Hospital Italiano de La Plata

🇦🇷

Ciudad De La Plata, Argentina

Sanatorio Allende

🇦🇷

Cordoba, Argentina

Hospital Privado-Universitario de Cordoba

🇦🇷

Cordoba, Argentina

Clinica Mayo de UMCB

🇦🇷

San Miguel de Tucuman, Argentina

Peter MacCallum Cancer Centre

🇦🇺

Melbourne, Australia

Royal Melbourne Hospital

🇦🇺

Melbourne, Australia

Westmead Hospital

🇦🇺

Westmead, Australia

AZ Sint-Jan

🇧🇪

Brugge, Belgium

Jules Bordet Institute

🇧🇪

Brussels, Belgium

UZ Brussel

🇧🇪

Brussels, Belgium

UZ Gent

🇧🇪

Gent, Belgium

UZ Leuven

🇧🇪

Leuven, Belgium

CHU de Liege

🇧🇪

Liege, Belgium

Jessa Ziekenhuis

🇧🇪

Limburg, Belgium

Fundacao Pio XII

🇧🇷

Barretos, Brazil

Universidade Federal De Minas Gerais - Hospital das Clínicas

🇧🇷

Belo Horizonte, Brazil

Centro de Pesquisa e Ensino em Oncologia de Santa Catarina CEPEN

🇧🇷

Florianopolis, Brazil

Universidade Federal do Ceara Hospital Universitario Walter Cantidio

🇧🇷

Fortaleza, Brazil

Fundacao Doutor Amaral Carvalho

🇧🇷

Jau, Brazil

Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto Hospital de Base

🇧🇷

Sao Jose do Rio Preto, Brazil

Real e Benemerita Associacao Portuguesa de Beneficencia

🇧🇷

Sao Paulo, Brazil

Fundacao Antonio Prudente A C Camargo Cancer Center

🇧🇷

Sao Paulo, Brazil

Sociedade Beneficente de Senhoras - Hospital Sírio Libanês

🇧🇷

São Paulo, Brazil

UMHAT 'Sveti Georgi'-Plovdiv

🇧🇬

Plovdiv, Bulgaria

Specialized Hospital for Active Treatment of Haematologic Diseases

🇧🇬

Sofia, Bulgaria

Multiprofile Hospital for Active Treatment 'Sveta Marina' EAD

🇧🇬

Varna, Bulgaria

Hôpital d'Instruction des Armées Percy

🇫🇷

Clamart, France

Institut Universitaire du Cancer Toulouse - Oncopole

🇫🇷

Toulouse, France

Rambam Medical Center

🇮🇱

Haifa, Israel

Hadassah Medical Center

🇮🇱

Jerusalem, Israel

Sheba Medical Center

🇮🇱

Ramat Gan, Israel

Sourasky (Ichilov) Medical Center

🇮🇱

Tel Aviv, Israel

Ospedale San Raffaele HSR Istituto Scientifico Universitario San Raffaele

🇮🇹

Milano, Italy

Ematologia Fondazione Univ. Policlinico Gemelli Università Cattolica del Sacro Cuore

🇮🇹

Roma, Italy

Akita University Hospital

🇯🇵

Akita, Japan

Chiba University Hospital

🇯🇵

Chiba, Japan

Tokai University Hospital

🇯🇵

Isehara, Japan

Japanese Red Cross Society Nagano Hospital

🇯🇵

Nagano, Japan

Okayama University Hospital

🇯🇵

Okayama, Japan

The Catholic University of Korea Seoul St Mary s Hospital

🇰🇷

Seoul, Korea, Republic of

Hospital Ampang

🇲🇾

Ampang Jaya, Malaysia

Penang General Hospital

🇲🇾

Georgetown, Malaysia

University Malaya Medical Centre

🇲🇾

Kuala Lumpur, Malaysia

Sunway Medical Centre

🇲🇾

Petaling Jaya, Malaysia

Leiden University Medical Center

🇳🇱

Leiden, Netherlands

Hosp Univ Vall D Hebron

🇪🇸

Barcelona, Spain

University Of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

University Hospitals Cleveland Medical Center

🇺🇸

Cleveland, Ohio, United States

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Baylor Scott and White Research Institute

🇺🇸

Dallas, Texas, United States

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