Trial Outcomes & Findings for NNITS-Nitazoxanide for Norovirus in Transplant Patients Study (NCT NCT03395405)

NCT ID: NCT03395405

Last Updated: 2025-05-21

Results Overview

Time (in days) from randomization until the study day when clinical resolution occurred. Clinical resolution was assessed from participant's daily diaries and was defined as cessation of vomiting and no stools classified by the Bristol Stool Chart as diarrhea (Type 6 or 7) for at least 48 hours.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

31 participants

Primary outcome timeframe

48 hours through Day 180

Results posted on

2025-05-21

Participant Flow

Participant milestones

Participant milestones
Measure
Nitazoxanide (500 mg)
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days. Nitazoxanide: a synthetic antiprotozoal agent, chemically designated as 2-acetyloxy-N-(5-nitro-2-thiazolyl) benzamide. Nitazoxanide will be supplied as 500 mg round, yellow, film-coated tablets.
Placebo
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days. Placebo: a round, yellow, film-coated tablet with the same inactive ingredients as the nitazoxanide tablet. The placebo tablet will be formulated for the same appearance as nitazoxanide.
Overall Study
STARTED
16
15
Overall Study
COMPLETED
12
10
Overall Study
NOT COMPLETED
4
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Nitazoxanide (500 mg)
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days. Nitazoxanide: a synthetic antiprotozoal agent, chemically designated as 2-acetyloxy-N-(5-nitro-2-thiazolyl) benzamide. Nitazoxanide will be supplied as 500 mg round, yellow, film-coated tablets.
Placebo
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days. Placebo: a round, yellow, film-coated tablet with the same inactive ingredients as the nitazoxanide tablet. The placebo tablet will be formulated for the same appearance as nitazoxanide.
Overall Study
Death
0
2
Overall Study
Lost to Follow-up
0
1
Overall Study
Protocol Violation
0
1
Overall Study
Withdrawal by Subject
3
1
Overall Study
COVID-19 Pandemic
1
0

Baseline Characteristics

NNITS-Nitazoxanide for Norovirus in Transplant Patients Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Total
n=31 Participants
Total of all reporting groups
Age, Continuous
58.9 years
STANDARD_DEVIATION 10.7 • n=99 Participants
55.8 years
STANDARD_DEVIATION 14.0 • n=107 Participants
57.4 years
STANDARD_DEVIATION 12.3 • n=206 Participants
Sex: Female, Male
Female
7 Participants
n=99 Participants
8 Participants
n=107 Participants
15 Participants
n=206 Participants
Sex: Female, Male
Male
9 Participants
n=99 Participants
7 Participants
n=107 Participants
16 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=99 Participants
1 Participants
n=107 Participants
3 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
13 Participants
n=99 Participants
12 Participants
n=107 Participants
25 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=99 Participants
2 Participants
n=107 Participants
3 Participants
n=206 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Black or African American
4 Participants
n=99 Participants
2 Participants
n=107 Participants
6 Participants
n=206 Participants
Race (NIH/OMB)
White
11 Participants
n=99 Participants
13 Participants
n=107 Participants
24 Participants
n=206 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=99 Participants
0 Participants
n=107 Participants
1 Participants
n=206 Participants
Duration of symptoms
Acute
4 Participants
n=99 Participants
3 Participants
n=107 Participants
7 Participants
n=206 Participants
Duration of symptoms
Chronic
12 Participants
n=99 Participants
12 Participants
n=107 Participants
24 Participants
n=206 Participants
Transplant type
Solid organ
15 Participants
n=99 Participants
15 Participants
n=107 Participants
30 Participants
n=206 Participants
Transplant type
Hematopoietic Stem Cell Transplant
1 Participants
n=99 Participants
0 Participants
n=107 Participants
1 Participants
n=206 Participants

PRIMARY outcome

Timeframe: 48 hours through Day 180

Population: Modified Intention-to-Treat (mITT) Population: The mITT population included all randomized participants who received at least one dose of assigned study drug. Analyses were performed according to randomized treatment assignment.

Time (in days) from randomization until the study day when clinical resolution occurred. Clinical resolution was assessed from participant's daily diaries and was defined as cessation of vomiting and no stools classified by the Bristol Stool Chart as diarrhea (Type 6 or 7) for at least 48 hours.

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Time to Initial Clinical Resolution of Norovirus Symptoms
19 days
Interval 1.0 to 31.0
11 days
Interval 2.0 to 14.0

SECONDARY outcome

Timeframe: Day 1 (baseline) through Day 60

Population: Safety Population: The safety population includes all participants who were randomized and received at least one dose of study treatment. Analyses are performed according to treatment received.

Participants experiencing at least one new laboratory adverse event. Laboratory parameters include White Blood Cell (WBC), Hemoglobin, Platelet Count, Creatinine, Alkaline Phosphatase, Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Blood Urea Nitrogen (BUN), and Bilirubin. Laboratory results were considered AEs using the following thresholds : WBC greater than the upper limit of normal (ULN), hemoglobin less than the lower limit of normal (LLN), platelet count less than the LLN; creatinine greater than the ULN; alkaline phosphatase greater than the ULN; ALT greater than the ULN, AST greater than the ULN, BUN greater than or equal to the ULN, and bilirubin greater than the ULN. ULN and LLN values differed by site, sex, and age category.

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Number of Participants Experiencing Laboratory Adverse Events (AEs)
11 Participants
13 Participants

SECONDARY outcome

Timeframe: Day 1 (baseline) and Day 180

Population: Modified Intention-to-Treat (mITT) Population: The mITT population included all randomized participants who received at least one dose of assigned study drug. Only participants with results at Days 1 and 180 were included. No participants in Placebo GI subgroup had results at both days. Analyses were performed based on randomized treatment assignment. Fewer than 5 participants per treatment arm tested positive for Norovirus GI at baseline thus no GI specific statistical analyses were conducted.

Change in viral titer defined as the difference between the Day 180 viral titer and the Day 1 viral titer. Participants were analyzed for the viral load test type (Norovirus GII or Norovirus GI) that they tested positive for at baseline (Day 1).

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=11 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=9 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Change in Viral Titer (Day 1 to Day 180)
Norovirus GII
-3.9 titer
Standard Deviation 9.8
-4.2 titer
Standard Deviation 6.8
Change in Viral Titer (Day 1 to Day 180)
Norovirus GI
-11.4 titer
Standard Deviation 3.5

SECONDARY outcome

Timeframe: Day 1 (baseline) through Day 60

Population: Safety Population: The safety population includes all participants who were randomized and received at least one dose of study treatment. Analyses are performed according to treatment received.

Hospitalizations included any admission to a hospital for treatment and were not reported as Serious Adverse Events (SAEs).

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Number of Participants Reporting Hospitalization
4 Participants
7 Participants

SECONDARY outcome

Timeframe: Day 1 (baseline) through Day 60

Population: Safety Population: The safety population includes all participants who were randomized and received at least one dose of study treatment. Analyses are performed according to treatment received.

Protocol-specified SAEs included any adverse event or suspected adverse reaction which, in the view of the investigator or sponsor, resulted in any of the following: death, life threatening adverse event, persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life function, congenital anomaly or birth defect, or an important medical event that may jeopardize the participant and require medical or surgical intervention. Hospitalizations were collected as a secondary outcome measure and were not reported as SAEs.

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Number of Participants Reporting Protocol-Specified SAEs
0 Participants
2 Participants

SECONDARY outcome

Timeframe: Day 1 (baseline) through Day 60

Population: Unsolicited adverse events were defined as any non-serious clinical adverse events that were not collected as clinical outcome measures and resulted in either modification in the administration of study drug or discontinuation of the study drug.

Unsolicited adverse events were defined as any non-serious clinical adverse events that were not collected as clinical outcome measures and resulted in either modification in the administration of study drug or discontinuation of the study drug.

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=16 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Number of Participants Experiencing Unsolicited Non-Serious Adverse Events
2 Participants
0 Participants

SECONDARY outcome

Timeframe: Day 1 (baseline) and Day 180

Population: Modified Intention-to-Treat (mITT) Population: The mITT population included all randomized participants who received at least one dose of assigned study drug. Only participants who tested positive at Day 1 were included. Analyses were performed based on randomized treatment assignment. Fewer than 5 participants per treatment arm tested positive for Norovirus GI at baseline thus no GI specific statistical analyses were conducted.

Time (in days) from randomization until the first study day the participant had either a negative result or a result less than the lower limit of quantitation (LLOQ) for the viral load test type (Norovirus GII or Norovirus GI) that they initially tested positive for at baseline. Participants were analyzed for the viral load test type (Norovirus GII or Norovirus GI) that they tested positive for at baseline (Day 1).

Outcome measures

Outcome measures
Measure
Nitazoxanide (500 mg)
n=14 Participants
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 Participants
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Time to First Negative Viral Load
Norovirus GI
NA days
Interval 12.0 to
Estimates were not calculable due to an insufficient number of participants (less than 50% of participants/treatment group) having a negative viral load.
22 days
Interval 13.0 to
Estimates were not calculable due to an insufficient number of participants (less than 50% of participants/treatment group) having a negative viral load.
Time to First Negative Viral Load
Norovirus GII
NA days
Interval 45.0 to
Estimates were not calculable due to an insufficient number of participants (less than 50% of participants/treatment group) having a negative viral load.
NA days
Interval 120.0 to
Estimates were not calculable due to an insufficient number of participants (less than 50% of participants/treatment group) having a negative viral load.

Adverse Events

Nitazoxanide (500 mg)

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Placebo

Serious events: 2 serious events
Other events: 13 other events
Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure
Nitazoxanide (500 mg)
n=16 participants at risk
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 participants at risk
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Respiratory, thoracic and mediastinal disorders
Hypoxic Respiratory Failure
0.00%
0/16 • 180 days
6.7%
1/15 • Number of events 1 • 180 days
Renal and urinary disorders
Uremia
0.00%
0/16 • 180 days
6.7%
1/15 • Number of events 1 • 180 days

Other adverse events

Other adverse events
Measure
Nitazoxanide (500 mg)
n=16 participants at risk
A single 500 mg tablet of nitazoxanide administered orally twice daily with food for 56 consecutive doses over 28 days.
Placebo
n=15 participants at risk
A single matching placebo tablet administered orally twice daily with food for 56 consecutive doses over 28 days.
Gastrointestinal disorders
Abdominal Pain
6.2%
1/16 • Number of events 1 • 180 days
0.00%
0/15 • 180 days
Investigations
Alanine Aminotransferase Increased
12.5%
2/16 • Number of events 2 • 180 days
6.7%
1/15 • Number of events 2 • 180 days
Investigations
Aspartate Aminotransferase Increased
12.5%
2/16 • Number of events 2 • 180 days
20.0%
3/15 • Number of events 4 • 180 days
Investigations
Blood Alkaline Phosphatase Increased
12.5%
2/16 • Number of events 4 • 180 days
20.0%
3/15 • Number of events 6 • 180 days
Investigations
Blood Bilirubin Increased
6.2%
1/16 • Number of events 1 • 180 days
13.3%
2/15 • Number of events 4 • 180 days
Investigations
Blood Creatinine Increased
37.5%
6/16 • Number of events 10 • 180 days
20.0%
3/15 • Number of events 3 • 180 days
Investigations
Blood Urea Increased
43.8%
7/16 • Number of events 13 • 180 days
20.0%
3/15 • Number of events 6 • 180 days
Investigations
Haemoglobin Decreased
18.8%
3/16 • Number of events 3 • 180 days
33.3%
5/15 • Number of events 6 • 180 days
Investigations
Platelet Count Decreased
18.8%
3/16 • Number of events 3 • 180 days
0.00%
0/15 • 180 days
Investigations
White Blood Cell Count Decreased
6.2%
1/16 • Number of events 1 • 180 days
20.0%
3/15 • Number of events 3 • 180 days
Metabolism and nutrition disorders
Hyperkalaemia
6.2%
1/16 • Number of events 1 • 180 days
0.00%
0/15 • 180 days
Investigations
White Blood Cell Count Increased
0.00%
0/16 • 180 days
13.3%
2/15 • Number of events 3 • 180 days

Additional Information

Michael G Ison

Northwestern University Feinberg School of Medicine

Phone: 3126954186

Results disclosure agreements

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

Restriction type: LTE60