Trial Outcomes & Findings for Presatovir in Hematopoietic Cell Transplant Recipients With Respiratory Syncytial Virus Infection of the Upper Respiratory Tract (NCT NCT02254408)
NCT ID: NCT02254408
Last Updated: 2018-08-06
Results Overview
The time-weighted average change, often referred to as the DAVG, provides the average viral burden change from baseline. The mean values presented were calculated using the ANCOVA model and are adjusted for baseline value and stratification factor.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
189 participants
Primary outcome timeframe
Baseline; Day 9
Results posted on
2018-08-06
Participant Flow
Participants were enrolled at study centers in North America, Europe, Australia and Asia. The first participant was screened on 23 January 2015 and the last study visit occurred on 14 July 2017.
213 participants were screened.
Participant milestones
| Measure |
Presatovir
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via nasogastric (NG) tube on Days 1, 5, 9, 13, and 17
|
Placebo
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Overall Study
STARTED
|
96
|
93
|
|
Overall Study
COMPLETED
|
88
|
83
|
|
Overall Study
NOT COMPLETED
|
8
|
10
|
Reasons for withdrawal
| Measure |
Presatovir
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via nasogastric (NG) tube on Days 1, 5, 9, 13, and 17
|
Placebo
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Overall Study
Randomized But Not Treated
|
1
|
3
|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Death
|
2
|
3
|
|
Overall Study
Investigator's Discretion
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
3
|
3
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
Baseline Characteristics
Participants in the Safety Analysis Set with available data were analyzed.
Baseline characteristics by cohort
| Measure |
Presatovir
n=95 Participants
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=90 Participants
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Total
n=185 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
52.1 years
STANDARD_DEVIATION 12.21 • n=95 Participants
|
51.4 years
STANDARD_DEVIATION 14.58 • n=90 Participants
|
51.8 years
STANDARD_DEVIATION 13.39 • n=185 Participants
|
|
Sex: Female, Male
Female
|
40 Participants
n=95 Participants
|
35 Participants
n=90 Participants
|
75 Participants
n=185 Participants
|
|
Sex: Female, Male
Male
|
55 Participants
n=95 Participants
|
55 Participants
n=90 Participants
|
110 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Asian
|
13 Participants
n=95 Participants
|
9 Participants
n=90 Participants
|
22 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
6 Participants
n=95 Participants
|
3 Participants
n=90 Participants
|
9 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
White
|
66 Participants
n=95 Participants
|
70 Participants
n=90 Participants
|
136 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=95 Participants
|
0 Participants
n=90 Participants
|
2 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Not Permitted
|
6 Participants
n=95 Participants
|
9 Participants
n=90 Participants
|
15 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
8 Participants
n=95 Participants
|
6 Participants
n=90 Participants
|
14 Participants
n=185 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
81 Participants
n=95 Participants
|
75 Participants
n=90 Participants
|
156 Participants
n=185 Participants
|
|
Region of Enrollment
Singapore
|
3 participants
n=95 Participants
|
2 participants
n=90 Participants
|
5 participants
n=185 Participants
|
|
Region of Enrollment
United States
|
58 participants
n=95 Participants
|
55 participants
n=90 Participants
|
116 participants
n=185 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=95 Participants
|
2 participants
n=90 Participants
|
5 participants
n=185 Participants
|
|
Region of Enrollment
Switzerland
|
0 participants
n=95 Participants
|
1 participants
n=90 Participants
|
1 participants
n=185 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=95 Participants
|
5 participants
n=90 Participants
|
8 participants
n=185 Participants
|
|
Region of Enrollment
South Korea
|
5 participants
n=95 Participants
|
1 participants
n=90 Participants
|
6 participants
n=185 Participants
|
|
Region of Enrollment
Israel
|
10 participants
n=95 Participants
|
11 participants
n=90 Participants
|
21 participants
n=185 Participants
|
|
Region of Enrollment
Australia
|
6 participants
n=95 Participants
|
3 participants
n=90 Participants
|
9 participants
n=185 Participants
|
|
Region of Enrollment
France
|
7 participants
n=95 Participants
|
10 participants
n=90 Participants
|
18 participants
n=185 Participants
|
|
Nasal Viral Load
|
6.31 log10 copies/mL
STANDARD_DEVIATION 1.899 • n=95 Participants • Participants in the Safety Analysis Set with available data were analyzed.
|
6.51 log10 copies/mL
STANDARD_DEVIATION 1.437 • n=88 Participants • Participants in the Safety Analysis Set with available data were analyzed.
|
6.41 log10 copies/mL
STANDARD_DEVIATION 1.691 • n=183 Participants • Participants in the Safety Analysis Set with available data were analyzed.
|
PRIMARY outcome
Timeframe: Baseline; Day 9Population: Full Analysis Set: participants who received at least 1 full dose of study drug and had an RSV viral load greater than or equal to the lower limit of quantification of the quantitative real-time polymerase chain reaction (RT-qPCR) assay in the Day 1 nasal sample, as determined by RT-qPCR.
The time-weighted average change, often referred to as the DAVG, provides the average viral burden change from baseline. The mean values presented were calculated using the ANCOVA model and are adjusted for baseline value and stratification factor.
Outcome measures
| Measure |
Presatovir
n=89 Participants
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=87 Participants
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Time-Weighted Average Change in Nasal Respiratory Syncytial Virus (RSV ) Viral Load From Baseline (Day 1) to Day 9
|
-1.26 log10 copies/mL
Standard Deviation 0.964
|
-0.91 log10 copies/mL
Standard Deviation 1.145
|
PRIMARY outcome
Timeframe: Up to Day 28Population: Full Analysis Set
A Lower Respiratory Tract Complication (LRTC) was defined as one of the below as determined by the adjudication committee: * Primary RSV lower respiratory tract infection (LRTI) * Secondary bacterial LRTI * LRTI due to unusual pathogens * Lower respiratory tract complication of unknown etiology
Outcome measures
| Measure |
Presatovir
n=89 Participants
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=87 Participants
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Percentage of Participants Who Developed a Lower Respiratory Tract Complication
|
11.2 percentage of participants
Interval 5.5 to 19.7
|
19.5 percentage of participants
Interval 11.8 to 29.4
|
SECONDARY outcome
Timeframe: Up to Day 28Population: Full Analysis Set
Participants were considered to have an event if either condition is met: * Participant develops a respiratory failure (of any cause) requiring mechanical ventilation (invasive or noninvasive) or; * Participant dies prior to or on Day 28
Outcome measures
| Measure |
Presatovir
n=89 Participants
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=87 Participants
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Percentage of Participants Who Developed Respiratory Failure (of Any Cause) Requiring Mechanical Ventilation (Invasive or Noninvasive) or All-cause Mortality
|
5.6 percentage of participants
Interval 1.8 to 12.6
|
5.7 percentage of participants
Interval 1.9 to 12.9
|
Adverse Events
Presatovir
Serious events: 18 serious events
Other events: 49 other events
Deaths: 2 deaths
Placebo
Serious events: 23 serious events
Other events: 47 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Presatovir
n=95 participants at risk
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=90 participants at risk
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Cardiac disorders
Pericardial effusion
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Gastrointestinal disorders
Vomiting
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
General disorders
Pyrexia
|
4.2%
4/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Immune system disorders
Food allergy
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Immune system disorders
Graft versus host disease in gastrointestinal tract
|
2.1%
2/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Clostridial sepsis
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Clostridium difficile colitis
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Cytomegalovirus infection
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Gastroenteritis norovirus
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Pneumonia
|
3.2%
3/95 • Up to 28 days
Safety Analysis Set
|
6.7%
6/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Pneumonia bacterial
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Pneumonia respiratory syncytial viral
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Pneumonia viral
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
2.2%
2/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Respiratory syncytial virus infection
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Sepsis
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Sepsis syndrome
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Septic shock
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
2.2%
2/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Infections and infestations
Upper respiratory tract infection
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Metabolism and nutrition disorders
Dehydration
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia recurrent
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Ataxia
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Dizziness postural
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Haemorrhage intracranial
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Somnolence
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Psychiatric disorders
Delirium
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
3.3%
3/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Acute lung injury
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Vascular disorders
Hypotension
|
1.1%
1/95 • Up to 28 days
Safety Analysis Set
|
0.00%
0/90 • Up to 28 days
Safety Analysis Set
|
Other adverse events
| Measure |
Presatovir
n=95 participants at risk
Presatovir 200 mg (4 x 50 mg tablets) administered as a single dose, orally or via NG tube on Days 1, 5, 9, 13, and 17
|
Placebo
n=90 participants at risk
Placebo administered orally or via NG tube on Days 1, 5, 9, 13, and 17
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
5.3%
5/95 • Up to 28 days
Safety Analysis Set
|
1.1%
1/90 • Up to 28 days
Safety Analysis Set
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.1%
2/95 • Up to 28 days
Safety Analysis Set
|
5.6%
5/90 • Up to 28 days
Safety Analysis Set
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.2%
3/95 • Up to 28 days
Safety Analysis Set
|
5.6%
5/90 • Up to 28 days
Safety Analysis Set
|
|
Gastrointestinal disorders
Diarrhoea
|
15.8%
15/95 • Up to 28 days
Safety Analysis Set
|
15.6%
14/90 • Up to 28 days
Safety Analysis Set
|
|
Gastrointestinal disorders
Nausea
|
13.7%
13/95 • Up to 28 days
Safety Analysis Set
|
11.1%
10/90 • Up to 28 days
Safety Analysis Set
|
|
Gastrointestinal disorders
Vomiting
|
10.5%
10/95 • Up to 28 days
Safety Analysis Set
|
13.3%
12/90 • Up to 28 days
Safety Analysis Set
|
|
General disorders
Asthenia
|
3.2%
3/95 • Up to 28 days
Safety Analysis Set
|
7.8%
7/90 • Up to 28 days
Safety Analysis Set
|
|
General disorders
Pyrexia
|
10.5%
10/95 • Up to 28 days
Safety Analysis Set
|
10.0%
9/90 • Up to 28 days
Safety Analysis Set
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.4%
7/95 • Up to 28 days
Safety Analysis Set
|
6.7%
6/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Dizziness
|
7.4%
7/95 • Up to 28 days
Safety Analysis Set
|
3.3%
3/90 • Up to 28 days
Safety Analysis Set
|
|
Nervous system disorders
Headache
|
5.3%
5/95 • Up to 28 days
Safety Analysis Set
|
7.8%
7/90 • Up to 28 days
Safety Analysis Set
|
|
Renal and urinary disorders
Acute kidney injury
|
3.2%
3/95 • Up to 28 days
Safety Analysis Set
|
5.6%
5/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.3%
6/95 • Up to 28 days
Safety Analysis Set
|
4.4%
4/90 • Up to 28 days
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
9.5%
9/95 • Up to 28 days
Safety Analysis Set
|
3.3%
3/90 • Up to 28 days
Safety Analysis Set
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.2%
4/95 • Up to 28 days
Safety Analysis Set
|
5.6%
5/90 • Up to 28 days
Safety Analysis Set
|
Additional Information
Gilead Clinical Study Information Center
Gilead Sciences
Phone: 1-833-445-3230 (GILEAD-0)
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met: * The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or * The study has been completed at all study sites for at least 2 years
- Publication restrictions are in place
Restriction type: OTHER