Trial Outcomes & Findings for Efficacy and Safety of Nitazoxanide in the Treatment of Colds Due to Enterovirus/Rhinovirus Infection (NCT NCT03605862)

NCT ID: NCT03605862

Last Updated: 2022-04-14

Results Overview

Subjects used the FLU-PRO questionnaire once daily in the evening to score the severity of 32 FLU-PRO symptoms. Symptom response was deemed achieved when the rating for each of the 32 FLU-PRO symptoms was ≤ its assigned threshold for 2 consecutive daily diary periods without use of symptom relief medication. The symptom response thresholds were developed by applying an algorithm to blinded symptoms data to select the set of 32 symptom thresholds most closely associated with patient-reported usual health.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

1756 participants

Primary outcome timeframe

Up to 21 days

Results posted on

2022-04-14

Participant Flow

Participant milestones

Participant milestones
Measure
Nitazoxanide
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
Two placebo tablets orally twice daily for 5 days
Overall Study
STARTED
872
884
Overall Study
COMPLETED
844
858
Overall Study
NOT COMPLETED
28
26

Reasons for withdrawal

Reasons for withdrawal
Measure
Nitazoxanide
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
Two placebo tablets orally twice daily for 5 days
Overall Study
Withdrawal by Subject
12
9
Overall Study
Adverse Event
2
1
Overall Study
Physician Decision
3
1
Overall Study
"Other" reasons included failure to attend follow up visits or inability to use the eDiary.
11
15

Baseline Characteristics

Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Nitazoxanide
n=872 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=884 Participants
Two placebo tablets orally twice daily for 5 days
Total
n=1756 Participants
Total of all reporting groups
Age, Continuous
37.1 years
STANDARD_DEVIATION 14.82 • n=872 Participants
37.3 years
STANDARD_DEVIATION 14.38 • n=884 Participants
37.2 years
STANDARD_DEVIATION 14.60 • n=1756 Participants
Sex: Female, Male
Female
613 Participants
n=872 Participants
567 Participants
n=884 Participants
1180 Participants
n=1756 Participants
Sex: Female, Male
Male
259 Participants
n=872 Participants
317 Participants
n=884 Participants
576 Participants
n=1756 Participants
Race/Ethnicity, Customized
Black or African American
173 Participants
n=872 Participants
175 Participants
n=884 Participants
348 Participants
n=1756 Participants
Race/Ethnicity, Customized
Hispanic
124 Participants
n=872 Participants
150 Participants
n=884 Participants
274 Participants
n=1756 Participants
Race/Ethnicity, Customized
White
551 Participants
n=872 Participants
536 Participants
n=884 Participants
1087 Participants
n=1756 Participants
Race/Ethnicity, Customized
Other
24 Participants
n=872 Participants
23 Participants
n=884 Participants
47 Participants
n=1756 Participants
Height
167.0 cm
STANDARD_DEVIATION 9.83 • n=872 Participants
168.3 cm
STANDARD_DEVIATION 10.38 • n=884 Participants
168.0 cm
STANDARD_DEVIATION 10.11 • n=1756 Participants
Weight
85.6 kg
STANDARD_DEVIATION 25.03 • n=872 Participants
85.8 kg
STANDARD_DEVIATION 22.74 • n=884 Participants
85.7 kg
STANDARD_DEVIATION 23.90 • n=1756 Participants
BMI
30.3 kg/m^2
STANDARD_DEVIATION 8.05 • n=872 Participants
30.3 kg/m^2
STANDARD_DEVIATION 8.09 • n=884 Participants
30.3 kg/m^2
STANDARD_DEVIATION 8.07 • n=1756 Participants
Smoking Status
Current Smoker
166 Participants
n=872 Participants
149 Participants
n=884 Participants
315 Participants
n=1756 Participants
Smoking Status
Past Smoker
120 Participants
n=872 Participants
123 Participants
n=884 Participants
243 Participants
n=1756 Participants
Smoking Status
Never Smoked
586 Participants
n=872 Participants
612 Participants
n=884 Participants
1198 Participants
n=1756 Participants
Time from Onset of Symptoms at First Study Drug Intake (hours)
26.6 hours
STANDARD_DEVIATION 9.1 • n=872 Participants
26.3 hours
STANDARD_DEVIATION 9.1 • n=884 Participants
26.5 hours
STANDARD_DEVIATION 9.1 • n=1756 Participants
Respiratory Infection(s)
Adenovirus
2 Participants
n=872 Participants
8 Participants
n=884 Participants
10 Participants
n=1756 Participants
Respiratory Infection(s)
Coronavirus (NL63, HKU1, 229E, or OC43)
63 Participants
n=872 Participants
54 Participants
n=884 Participants
117 Participants
n=1756 Participants
Respiratory Infection(s)
Enterovirus/Rhinovirus
286 Participants
n=872 Participants
298 Participants
n=884 Participants
584 Participants
n=1756 Participants
Respiratory Infection(s)
Human Metapneumovirus
5 Participants
n=872 Participants
6 Participants
n=884 Participants
11 Participants
n=1756 Participants
Respiratory Infection(s)
Influenza A
6 Participants
n=872 Participants
3 Participants
n=884 Participants
9 Participants
n=1756 Participants
Respiratory Infection(s)
Mycoplasma pneumoniae
0 Participants
n=872 Participants
1 Participants
n=884 Participants
1 Participants
n=1756 Participants
Respiratory Infection(s)
Parainfluenza
38 Participants
n=872 Participants
36 Participants
n=884 Participants
74 Participants
n=1756 Participants
Respiratory Infection(s)
Respiratory Syncytial Virus (RSV)
19 Participants
n=872 Participants
18 Participants
n=884 Participants
37 Participants
n=1756 Participants
Baseline Symptom Score, ITTI Population
1.4 mean of patient-reported symptom ratings
STANDARD_DEVIATION 0.59 • n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
1.4 mean of patient-reported symptom ratings
STANDARD_DEVIATION 0.59 • n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
1.4 mean of patient-reported symptom ratings
STANDARD_DEVIATION 0.59 • n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Patient-Reported Health Status, ITTI Population
At Usual Health
35 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
42 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
77 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Patient-Reported Health Status, ITTI Population
Not At Usual Health
251 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
256 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
507 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Illness Severity, ITTI Population
No cold symptoms
3 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
2 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
5 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Illness Severity, ITTI Population
Mild
40 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
34 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
74 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Illness Severity, ITTI Population
Moderate
147 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
149 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
296 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Illness Severity, ITTI Population
Severe
83 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
93 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
176 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Illness Severity, ITTI Population
Very severe
13 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
20 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
33 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Interference in Activities, ITTI Population
Not at all
48 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
37 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
85 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Interference in Activities, ITTI Population
A little bit
74 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
86 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
160 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Interference in Activities, ITTI Population
Somewhat
87 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
94 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
181 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Interference in Activities, ITTI Population
Quite a bit
58 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
59 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
117 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Interference in Activities, ITTI Population
Very much
19 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
22 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
41 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
Much better than yesterday
5 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
4 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
9 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
Somewhat better than yesterday
11 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
10 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
21 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
A little better than yesterday
24 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
20 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
44 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
About the same as yesterday
89 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
80 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
169 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
A little worse than yesterday
79 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
93 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
172 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
Somewhat worse than yesterday
47 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
48 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
95 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
Baseline Symptom Severity Compared to Yesterday, ITTI Population
Much worse than yesterday
31 Participants
n=286 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
43 Participants
n=298 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.
74 Participants
n=584 Participants • Analysis population is the primary efficacy (intent-to-treat-infected, ITTI) population with laboratory-confirmed EV/RV infection.

PRIMARY outcome

Timeframe: Up to 21 days

Population: The primary efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Subjects used the FLU-PRO questionnaire once daily in the evening to score the severity of 32 FLU-PRO symptoms. Symptom response was deemed achieved when the rating for each of the 32 FLU-PRO symptoms was ≤ its assigned threshold for 2 consecutive daily diary periods without use of symptom relief medication. The symptom response thresholds were developed by applying an algorithm to blinded symptoms data to select the set of 32 symptom thresholds most closely associated with patient-reported usual health.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Time From First Dose to Symptom Response Over 21 Days of Follow up Based Upon the FLU-PRO Instrument (Novel Endpoint)
122.5 hours
Interval 58.0 to 244.0
137.1 hours
Interval 75.0 to 243.0

SECONDARY outcome

Timeframe: Up to 21 days

Population: The efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Subjects completed a diary including rating ability to perform normal activities on a scale from 0 (able to perform no normal activities) to 10 (able to perform all normal activities) daily in the evening. The time from first dose to ability to perform all normal activities is the time in hours between the first dose of study medication and that time when the subject first reported a score of "10" (able to perform all normal activities) for two consecutive daily diary periods without use of symptom relief medication.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Time From First Dose to Ability to Perform All Normal Activities
174.3 hours
Interval 80.0 to 340.0
175.4 hours
Interval 105.0 to 318.0

SECONDARY outcome

Timeframe: 28 days

Population: The efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Complications of colds due to EV/RV infection include pneumonia, otitis media, bronchitis, sinusitis, exacerbations of asthma or COPD, worsening of pre-existing health conditions, secondary infections requiring systemic antibiotic use, hospitalization due to cold or complications of the cold, and death due to cold or complications of the cold. Proportions experiencing complications of EV/RV infection were compared across treatment groups.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Proportions Experiencing Complications of EV/RV Infection
12 Participants
20 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 21 days

Population: The efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Subjects completed the FLU-PRO questionnaire including global assessment questions daily in the evening. The time from first dose to ability to return to usual health is the time in hours from the first dose of study medication to the first time when the subject answered "Have you returned to your usual health?" with "yes" for two consecutive daily diary periods without the use of symptom relief medication.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Time to Return to Usual Health
154.1 hours
Interval 80.0 to 320.0
174.9 hours
Interval 104.0 to 345.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 2, 3, and 7

Population: The efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Proportion of subjects with nasopharyngeal swab collected testing positive for Enterovirus/Rhinovirus (EV/RV) infection by RT-PCR at each time point.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Proportion Positive for EV/RV by RT-PCR at Days 2, 3 and 7
Day 2
0.81 proportion of participants
0.81 proportion of participants
Proportion Positive for EV/RV by RT-PCR at Days 2, 3 and 7
Day 3
0.79 proportion of participants
0.79 proportion of participants
Proportion Positive for EV/RV by RT-PCR at Days 2, 3 and 7
Day 7
0.66 proportion of participants
0.76 proportion of participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 2, 3, and 7

Population: Analysis population includes subjects with laboratory-confirmed EV/RV who were positive for EV/RV at the preceding swab collection time point. Per protocol, day 2 and 3 visits were optional.

Changes from baseline to day 2, baseline to day 3, and baseline to day 7 in EV/RV virus titer measured by quantitative RT-PCR. Samples negative for EV/RV were assigned the value of the limit of detection for the RT-PCR assay.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Analysis of Change From Baseline to Days 2, 3 and 7 in EV/RV Virus Titer
Day 2
-0.2203 log10 RNA copies/mL
Standard Error 0.0934
-0.4186 log10 RNA copies/mL
Standard Error 0.0884
Analysis of Change From Baseline to Days 2, 3 and 7 in EV/RV Virus Titer
Day 3
-0.5577 log10 RNA copies/mL
Standard Error 0.1272
-0.8282 log10 RNA copies/mL
Standard Error 0.1053
Analysis of Change From Baseline to Days 2, 3 and 7 in EV/RV Virus Titer
Day 7
-1.5113 log10 RNA copies/mL
Standard Error 0.1373
-1.6470 log10 RNA copies/mL
Standard Error 0.1231

OTHER_PRE_SPECIFIED outcome

Timeframe: 21 days

Population: The intent-to-treat-infected (ITTI) population consisted of all subjects positive for EV/RV at Baseline. Per the Statistical Analysis Plan, the response definition misclassification rate was to be calculated prior to unblinding including all data for subjects in the ITTI population.

The proportion of patient diaries misclassified by the response definition used for the primary efficacy analysis compared to patient reported usual health. A diary was considered "misclassified" if the response definition predicted "responded" and the patient reported not being at usual health or if the response definition predicted "not responded" and the patient reported being at usual health.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=584 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
Two placebo tablets orally twice daily for 5 days
Response Misclassification Rate Compared to Usual Health
0.21915 n diaries misclassified/n diaries

POST_HOC outcome

Timeframe: 21 days

Population: The intent-to-treat-infected (ITTI) population consisted of all subjects positive for EV/RV at Baseline. Consistent with calculation of the response definition misclassification rate, the correlation coefficient was calculated for all subjects in the ITTI population regardless of treatment group assignment.

The correlation coefficient between sustained response and return to usual health was calculated for the pooled ITTI population (i.e., not by treatment group) as a measure of association between the primary endpoint response definition and its intended anchor, patient-reported return to usual health.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=584 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
Two placebo tablets orally twice daily for 5 days
Correlation Coefficient for Sustained Response and Return to Usual Health
0.43 correlation coefficient

POST_HOC outcome

Timeframe: 21 days

Population: Modified ITTI population consists of 387 subjects with laboratory-confirmed EV/RV infection and Baseline subject-reported assessment that symptoms are present, the symptoms are not consistent with the subject's usual health, the symptoms interfere with daily activities, and the symptoms have worsened or remained the same relative to the previous day. Assessment was completed via the Baseline FLU-PRO questionnaire.

Examination of Baseline disease characteristics revealed many subjects reporting via the Baseline FLU-PRO questionnaire that they are at their usual state of health, that symptoms do not interfere with any usual activities, and/or that symptoms are already improving. The pre-specified analysis of Time to Return for Usual Health was repeated for the population with Baseline subject-reported assessment that symptoms are present, the symptoms are not consistent with the subject's usual health, the symptoms interfere with daily activities, and the symptoms have worsened or remained the same relative to the previous day. Time to Return to Usual Health is the time in hours between the first dose of study medication and the first time at which the subject answered "Have you returned to your Usual Health today?" via the daily FLU-PRO questionnaire with a "yes" for two consecutive diary periods without use of symptom relief medication.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=183 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=204 Participants
Two placebo tablets orally twice daily for 5 days
Time to Return to Usual Health, Modified ITTI Population
153.7 hours
Interval 96.0 to 317.0
195.0 hours
Interval 123.0 to 365.0

POST_HOC outcome

Timeframe: 21 days

Population: The efficacy (intent-to-treat infected, ITTI) population consisted of 584 subjects (N=286 in NTZ group, N=298 in Placebo group) with laboratory-confirmed Enterovirus/Rhinovirus (EV/RV) infection.

Alternative means of endpoint construction were pursued to strengthen the relationship between symptoms-based endpoint measures and subject global assessments of health. Time to Sustained Clinical Recovery is an endpoint based on evidence of meaningful within-subject change sustained for the duration of the study. Time to Sustained Clinical Recovery is the time in hours from the first dose of study medication to the first time at which the subject reports a decrease in total FLU-PRO score from the previous diary with assessment that symptoms are at least "somewhat better than yesterday", no oral temperature ≥100.4 F in the prior 24 hours, and no future increase in any of the FLU-PRO domains except within validated background levels.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=286 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=298 Participants
Two placebo tablets orally twice daily for 5 days
Time to Sustained Clinical Recovery
171.4 hours
Interval 81.0 to 411.0
221.5 hours
Interval 103.0 to 504.0

POST_HOC outcome

Timeframe: 21 days

Population: Modified ITTI population consists of 387 subjects with laboratory-confirmed EV/RV infection and Baseline subject-reported assessment that symptoms are present, the symptoms are not consistent with the subject's usual health, the symptoms interfere with daily activities, and the symptoms have worsened or remained the same relative to the previous day. Assessment was completed via the Baseline FLU-PRO questionnaire.

Analysis of Time to Sustained Clinical Recovery was repeated for the population with Baseline subject-reported assessment that symptoms are present, the symptoms are not consistent with the subject's usual health, the symptoms interfere with daily activities, and the symptoms have worsened or remained the same relative to the previous day. Time to Sustained Clinical Recovery is the time in hours from the first dose of study medication to the first time at which the subject reports a decrease in total FLU-PRO score from the previous diary with assessment that symptoms are at least "somewhat better than yesterday", no oral temperature ≥100.4 F in the prior 24 hours, and no future increase in any of the FLU-PRO domains except within validated background levels.

Outcome measures

Outcome measures
Measure
Nitazoxanide
n=183 Participants
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=204 Participants
Two placebo tablets orally twice daily for 5 days
Time to Sustained Clinical Recovery, Modified ITTI Population
150.3 hours
Interval 81.0 to 322.0
244.1 hours
Interval 119.0 to 504.0

Adverse Events

Nitazoxanide

Serious events: 4 serious events
Other events: 200 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 58 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Nitazoxanide
n=872 participants at risk
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=884 participants at risk
Two placebo tablets orally twice daily for 5 days
Cardiac disorders
Cardiac failure congestive
0.11%
1/872 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.
0.00%
0/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Hepatobiliary disorders
Cholelithiasis
0.11%
1/872 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.
0.00%
0/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Infections and infestations
Gastroenteritis viral
0.11%
1/872 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.
0.00%
0/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Infections and infestations
Post procedural infection
0.11%
1/872 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.
0.00%
0/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Infections and infestations
Urosepsis
0.11%
1/872 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.
0.00%
0/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Nervous system disorders
Status epilepticus
0.00%
0/872 • 28 days or until resolution of all adverse events, whichever occurred later.
0.11%
1/884 • Number of events 1 • 28 days or until resolution of all adverse events, whichever occurred later.

Other adverse events

Other adverse events
Measure
Nitazoxanide
n=872 participants at risk
Two Nitazoxanide 300 mg tablets orally twice daily for 5 days
Placebo
n=884 participants at risk
Two placebo tablets orally twice daily for 5 days
Renal and urinary disorders
Chromaturia
17.5%
153/872 • 28 days or until resolution of all adverse events, whichever occurred later.
1.1%
10/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Gastrointestinal disorders
Diarrhoea
6.1%
53/872 • 28 days or until resolution of all adverse events, whichever occurred later.
4.3%
38/884 • 28 days or until resolution of all adverse events, whichever occurred later.
Gastrointestinal disorders
Nausea
2.8%
24/872 • 28 days or until resolution of all adverse events, whichever occurred later.
2.1%
19/884 • 28 days or until resolution of all adverse events, whichever occurred later.

Additional Information

Jessica Fulgencio

Romark Laboratories, L.C.

Phone: 813-282-8544

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place