Trial Outcomes & Findings for Staccato Granisetron® (AZ 010) for the Treatment of Cyclic Vomiting Syndrome (NCT NCT04645953)
NCT ID: NCT04645953
Last Updated: 2025-06-11
Results Overview
The primary efficacy endpoint for this study was the number vomiting/retching events in the two hours following initial treatment. Patients recorded the number of vomiting and retching events that they experienced which occurred 2 hours post dose. Observations occurred at 4 time points: 30 minutes post-dose, 1 hour post-dose, 90 minutes post-dose, and 2 hours post-dose. While each treatment group contained 47-49 patients, only 22-35 patients per treatment group (\~45-70%) recorded vomiting/retching events at any given time point. Safety and tolerability of AZ-010 was assessed by evaluating adverse events, vital signs, 12-lead ECG, clinical laboratory results, and physical examination. Clinically significant deteriorations in physical examination findings (in the opinion of the investigator) are captured and summarized as adverse events.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
150 participants
Primary outcome timeframe
Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.
Results posted on
2025-06-11
Participant Flow
This study was a multicenter study conducted at 18 sites in the United States. Of these, only 17 sites were activated, and only 15 sites enrolled subjects. Study Period: 1 year 5 months Study Start: 05 February 2021 Study End: 26 July 2022 50 patients randomized to each of 3 treatment groups: 1 mg AZ-010, 3 mg AZ-010, or matching Staccato® Placebo Patients were screened at the Research Clinic to determine eligibility for study participation no more than14 days prior to Visit 2.
Participant eligibility inclusion and exclusion criteria via the following assessments: Medical history (with particular reference to history of vomiting symptoms, CVS diagnosis, typical duration of CVS episode, average # of vomiting/retching events in typical CVS episode, frequency of CVS episodes per year, any triggers for CVS episode, conditions that may cause similar symptoms, treatment history, and current medications, medical history, COVID-19 screening.
Participant milestones
| Measure |
1mg AZ010
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
3mg AZ010
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
Placebo
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
|---|---|---|---|
|
Overall Study
STARTED
|
50
|
51
|
49
|
|
Overall Study
COMPLETED
|
48
|
48
|
46
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
3
|
Reasons for withdrawal
| Measure |
1mg AZ010
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
3mg AZ010
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
Placebo
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
|---|---|---|---|
|
Overall Study
Lost to Follow-up
|
2
|
1
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
0
|
|
Overall Study
Physician Decision
|
0
|
1
|
0
|
|
Overall Study
Discontinued Prior to Dosing
|
0
|
0
|
2
|
Baseline Characteristics
Staccato Granisetron® (AZ 010) for the Treatment of Cyclic Vomiting Syndrome
Baseline characteristics by cohort
| Measure |
1mg AZ010
n=50 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=51 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=49 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
Total
n=150 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
33.5 years
STANDARD_DEVIATION 11.95 • n=5 Participants
|
36.0 years
STANDARD_DEVIATION 12.30 • n=7 Participants
|
36.2 years
STANDARD_DEVIATION 10.22 • n=5 Participants
|
35.2 years
STANDARD_DEVIATION 11.53 • n=4 Participants
|
|
Sex: Female, Male
Female
|
34 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
104 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
46 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
41 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
127 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
41 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
130 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Height
|
168.85 cm
STANDARD_DEVIATION 9.166 • n=5 Participants
|
168.73 cm
STANDARD_DEVIATION 9.729 • n=7 Participants
|
168.81 cm
STANDARD_DEVIATION 7.910 • n=5 Participants
|
168.79 cm
STANDARD_DEVIATION 8.919 • n=4 Participants
|
|
Weight
|
83.28 kg
STANDARD_DEVIATION 24.854 • n=5 Participants
|
78.85 kg
STANDARD_DEVIATION 19.500 • n=7 Participants
|
82.08 kg
STANDARD_DEVIATION 26.443 • n=5 Participants
|
81.38 kg
STANDARD_DEVIATION 23.655 • n=4 Participants
|
|
Body Mass Index (kg/m2)
|
28.979 kg/m^2
STANDARD_DEVIATION 7.7236 • n=5 Participants
|
27.529 kg/m^2
STANDARD_DEVIATION 6.0793 • n=7 Participants
|
28.238 kg/m^2
STANDARD_DEVIATION 7.9544 • n=5 Participants
|
28.244 kg/m^2
STANDARD_DEVIATION 7.2635 • n=4 Participants
|
|
Childbearing potential (Yes)
|
24 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
80 Participants
n=4 Participants
|
|
Childbearing potential (No)
|
26 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
70 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.Population: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
The primary efficacy endpoint for this study was the number vomiting/retching events in the two hours following initial treatment. Patients recorded the number of vomiting and retching events that they experienced which occurred 2 hours post dose. Observations occurred at 4 time points: 30 minutes post-dose, 1 hour post-dose, 90 minutes post-dose, and 2 hours post-dose. While each treatment group contained 47-49 patients, only 22-35 patients per treatment group (\~45-70%) recorded vomiting/retching events at any given time point. Safety and tolerability of AZ-010 was assessed by evaluating adverse events, vital signs, 12-lead ECG, clinical laboratory results, and physical examination. Clinically significant deteriorations in physical examination findings (in the opinion of the investigator) are captured and summarized as adverse events.
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
Number Vomiting & Retching Events, First Available Efficacy Data, 30 minutes post dose
|
0.69 event
Standard Deviation 1.168
|
1.43 event
Standard Deviation 1.975
|
1.33 event
Standard Deviation 2.239
|
|
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
Number Vomiting & Retching Events First Available Efficacy Data, 90 minutes post dose
|
2.17 event
Standard Deviation 4.243
|
3.32 event
Standard Deviation 3.906
|
2.59 event
Standard Deviation 4.205
|
|
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
Number Vomiting & Retching Events First Available Efficacy Data, 1 hour post dose
|
1.48 event
Standard Deviation 2.694
|
2.43 event
Standard Deviation 3.099
|
2.13 event
Standard Deviation 3.180
|
|
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
Number Vomiting & Retching Events First Available Efficacy Data, 2 hours post dose
|
3.10 event
Standard Deviation 5.576
|
4.21 event
Standard Deviation 5.279
|
2.61 event
Standard Deviation 4.408
|
SECONDARY outcome
Timeframe: 24 hours after treatment dosePopulation: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
Assessment of anxiety/panic at 2, 6, 12, and 24 hours following treatment. The assessment of anxiety/panic Visual Analog Scale (VAS) score whereby 0 = no anxiety/panic and 100 = worst possible anxiety/panic 0-100; 0 = no anxiety/panic and 100 = worst possible anxiety/panic). Participants were asked to rate their anxiety/panic on scale of 0-100 at each time point. The higher the number, the more intense the symptom.
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Anxiety/Panic Visual Analog Scale (VAS) Score
Anxiety/Panic VAS Score, 12 hour, post-dose
|
31.00 score on a scale
Standard Deviation 28.827
|
51.15 score on a scale
Standard Deviation 31.947
|
64.00 score on a scale
Standard Deviation 26.533
|
|
Anxiety/Panic Visual Analog Scale (VAS) Score
Anxiety/Panic VAS Score, 2 hour, post-dose
|
51.30 score on a scale
Standard Deviation 34.907
|
52.08 score on a scale
Standard Deviation 35.020
|
47.72 score on a scale
Standard Deviation 32.849
|
|
Anxiety/Panic Visual Analog Scale (VAS) Score
Anxiety/Panic VAS Score, 6 hour, post-dose
|
48.25 score on a scale
Standard Deviation 17.343
|
64.00 score on a scale
Standard Deviation 35.071
|
51.29 score on a scale
Standard Deviation 20.605
|
|
Anxiety/Panic Visual Analog Scale (VAS) Score
Anxiety/Panic VAS Score, 24 hour, post-dose
|
18.00 score on a scale
Standard Deviation 19.378
|
37.88 score on a scale
Standard Deviation 31.439
|
62.25 score on a scale
Standard Deviation 30.478
|
SECONDARY outcome
Timeframe: 24 hours after each dosePopulation: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
The duration of the CVS episode at 24 hours in relation to their typical CVS episodes (Prior Episode Questionnaire) and the intensity of the CVS episode at 24 hours in relation to their typical CVS episodes. A 3-point scale was used. The questions were prompted approximately 24 hours after each dose of study medication was administered. Duration: (Score definition: 1 = shorter duration than typical previous episode; 2 = same duration as typical previous episode; 3 = longer duration than typical previous episode) Intensity: (Score definition: 1 = less intense than typical previous episode; 2 = same intensity as typical previous episode; 3 = more intense than typical previous episode)
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Prior Episode Questionnaire
Prior Episode Questionnaire, 24 Hours Post-Dose, Duration Of Current Episode
|
1.46 score on a scale
Standard Deviation 0.658
|
1.27 score on a scale
Standard Deviation 0.550
|
1.52 score on a scale
Standard Deviation 0.680
|
|
Prior Episode Questionnaire
Prior Episode Questionnaire, 24 Hours Post-Dose, Intensity Of Current Episode
|
1.58 score on a scale
Standard Deviation 0.654
|
1.55 score on a scale
Standard Deviation 0.671
|
1.43 score on a scale
Standard Deviation 0.598
|
SECONDARY outcome
Timeframe: 24 hours post dosePopulation: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
The percentage of patients who used rescue medication within the first day of treatment; patients confirming the individual had used some sort of rescue medication.
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Rescue Medication Use Within 1 Day of Dose
Percentage of patients confirming "Yes" response to rescue medication use, 2 hours post dose
|
3 Participants
|
2 Participants
|
1 Participants
|
|
Rescue Medication Use Within 1 Day of Dose
Percentage of patients confirming "Yes" response to rescue medication use, 24 hours post dose
|
9 Participants
|
11 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Within Day 1 after dosingPopulation: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office for care within the first day following dosing for a CVS episode.
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office
Patients requiring healthcare provider visit within 24 hours post dose of CVS episode.
|
2 Participants
|
0 Participants
|
0 Participants
|
|
Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office
Patients not requiring healthcare provider visit within 24 hours post dose of CVS episode.
|
45 Participants
|
49 Participants
|
44 Participants
|
SECONDARY outcome
Timeframe: Within 24 hours following treatment.Population: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
The Rhodes Index of nausea, vomiting, and retching (RINVR) at 6, 12, and 24 hours following treatment. The Rhodes Index of nausea, vomiting, and retching (RINVR) is designed to assess the degree of nausea distress and vomiting distress in patients. It is composed of 8 questions, and each question has 5 choices. The 5 choices for each individual question are scores from 0 to 4, with 0 being the lowest level without any symptoms related to nausea/vomiting/retching and 4 being the highest level. Individual question scores can be tracked over time, or a composite score with a total scoring range of 0 to 32 can be formed by adding the individual scores of the symptom occurrence and degree of discomfort questions together. Composite scores classified as follows: 0: no symptoms, 1-8: mild, 9-16: moderate, 17-24: great, 25-32: severe. This scale was adapted for the ePD, and patients were prompted to answer the questions according to the Schedule of Events.
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 6 hour, post-dose
|
11.20 score on a scale
Standard Deviation 7.741
|
11.85 score on a scale
Standard Deviation 7.893
|
11.79 score on a scale
Standard Deviation 7.695
|
|
Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 12 hour, post-dose
|
6.57 score on a scale
Standard Deviation 7.710
|
10.45 score on a scale
Standard Deviation 9.237
|
7.81 score on a scale
Standard Deviation 8.274
|
|
Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 24 hour, post-dose
|
6.76 score on a scale
Standard Deviation 7.096
|
8.87 score on a scale
Standard Deviation 8.471
|
7.92 score on a scale
Standard Deviation 8.057
|
SECONDARY outcome
Timeframe: Up to 24 hours post dose.Population: Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months.
The patient was asked the abdominal pain in relation to their last vomiting/retching episode, whereby 0 = no pain and 100 = worst possible pain at timepoints 2, 6, 12 and 24 hours post-dose
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Abdominal Pain, Visual Analog Scale (VAS) Score
Abdominal Pain VAS Score,2 hour, post-dose
|
45.48 units on a scale
Standard Deviation 27.318
|
53.04 units on a scale
Standard Deviation 32.227
|
51.60 units on a scale
Standard Deviation 29.390
|
|
Abdominal Pain, Visual Analog Scale (VAS) Score
Abdominal Pain VAS Score,6 hour, post-dose
|
51.50 units on a scale
Standard Deviation 17.849
|
47.25 units on a scale
Standard Deviation 33.970
|
32.57 units on a scale
Standard Deviation 22.846
|
|
Abdominal Pain, Visual Analog Scale (VAS) Score
Abdominal Pain VAS Score,12 hour, post-dose
|
36.67 units on a scale
Standard Deviation 7.024
|
51.38 units on a scale
Standard Deviation 33.393
|
61.25 units on a scale
Standard Deviation 30.489
|
|
Abdominal Pain, Visual Analog Scale (VAS) Score
Abdominal Pain VAS Score, 24 hour, post-dose
|
32.60 units on a scale
Standard Deviation 17.771
|
43.88 units on a scale
Standard Deviation 38.264
|
66.25 units on a scale
Standard Deviation 11.354
|
SECONDARY outcome
Timeframe: Within 24 hours post dose.Population: Per protocol population (CVS patients between 18 and 60 years of age who reported intensity of vomiting attack.
The Intensity of Attack scale assessed the severity of the last vomiting/retching episode at 2, 6, 12, and 24 hours post dose. Intensity of attack is evaluated on a scale of 1-4 (1=Mild, 2=Moderate, 3=Severe, 4=Excruciating).
Outcome measures
| Measure |
1mg AZ010
n=47 Participants
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
|
3mg AZ010
n=49 Participants
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
|
Placebo
n=48 Participants
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
|
|---|---|---|---|
|
Intensity of Vomiting/Retching Attack
Intensity of Attack Scale (Efficacy Population) 2 hours post-dose
|
2.29 units on a scale
Standard Deviation 0.488
|
3.00 units on a scale
Standard Deviation 0.894
|
2.25 units on a scale
Standard Deviation 0.957
|
|
Intensity of Vomiting/Retching Attack
Intensity of Attack Scale (Efficacy Population) 6 hour, post-dose
|
1.86 units on a scale
Standard Deviation 0.690
|
2.29 units on a scale
Standard Deviation 0.756
|
1.57 units on a scale
Standard Deviation 0.787
|
|
Intensity of Vomiting/Retching Attack
Intensity of Attack Scale (Efficacy Population) 12 hour, post-dose
|
1.33 units on a scale
Standard Deviation 0.577
|
2.31 units on a scale
Standard Deviation 0.947
|
1.75 units on a scale
Standard Deviation 0.957
|
|
Intensity of Vomiting/Retching Attack
Intensity of Attack Scale (Efficacy Population) 24 hour, post-dose
|
2.20 units on a scale
Standard Deviation 0.837
|
2.38 units on a scale
Standard Deviation 0.916
|
2.25 units on a scale
Standard Deviation 0.957
|
Adverse Events
1mg AZ010
Serious events: 3 serious events
Other events: 28 other events
Deaths: 0 deaths
3mg AZ010
Serious events: 3 serious events
Other events: 40 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 44 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
1mg AZ010
n=50 participants at risk
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
3mg AZ010
n=51 participants at risk
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
Placebo
n=49 participants at risk
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
|---|---|---|---|
|
Gastrointestinal disorders
CVS, Nausea
|
4.0%
2/50 • Number of events 2 • up to 30 days after the last dose
|
3.9%
2/51 • Number of events 2 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 2 • up to 30 days after the last dose
|
|
Surgical and medical procedures
Surgical and medical procedures
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Hepatobiliary disorders
Biliary dilatation
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Vascular disorders
Hypertension
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
Other adverse events
| Measure |
1mg AZ010
n=50 participants at risk
Single orally-inhaled dose
1mg AZ010: Subject will receive a single inhaled dose (1mg)
single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
3mg AZ010
n=51 participants at risk
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
Placebo
n=49 participants at risk
Single orally-inhaled dose
Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
|
|---|---|---|---|
|
Gastrointestinal disorders
Hematemesis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Indigestion
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Pain abdominal
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Retching
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Vomited
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Nervous system disorders
Headache
|
6.0%
3/50 • Number of events 4 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
8.2%
4/49 • Number of events 4 • up to 30 days after the last dose
|
|
Nervous system disorders
Burning sensation
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 2 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Nervous system disorders
Dizziness
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Nervous system disorders
Migraine
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Infections and infestations
COVID-19
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 2 • up to 30 days after the last dose
|
|
Infections and infestations
Acute diverticulitis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Infections and infestations
Acute pharyngitis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Infections and infestations
Acute sinusitis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Infections and infestations
Rhinovirus infection
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Infections and infestations
Yeast infection
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 2 • up to 30 days after the last dose
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Metabolism and nutrition disorders
Metabolic alkalosis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 3 • up to 30 days after the last dose
|
|
Psychiatric disorders
Panic attack
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Psychiatric disorders
Depersonalization
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 3 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Psychiatric disorders
Difficulty sleeping
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Psychiatric disorders
Stress
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 2 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Respiratory, thoracic and mediastinal disorders
Dry cough
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 2 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Respiratory, thoracic and mediastinal disorders
Nosebleed
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
General disorders
Fatigue
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
General disorders
Fever
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
General disorders
Pain
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
General disorders
Poikilothermia
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
General disorders
Tiredness
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle aches
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Musculoskeletal and connective tissue disorders
Muscle cramps
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Musculoskeletal and connective tissue disorders
Weakness in extremity
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Vascular disorders
Flushing
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Vascular disorders
Hypertension
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Vascular disorders
Hypotension
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Vascular disorders
Pale skin
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Investigations
Endoscopic retrograde cholangiopancreatography
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Investigations
Lactate increased
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Investigations
QT interval prolonged
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Investigations
ST segment depressed
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Investigations
Transaminitis
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Skin and subcutaneous tissue disorders
Redness
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Skin and subcutaneous tissue disorders
Sweating
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 2 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Cardiac disorders
Left anterior fascicular block
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Cardiac disorders
Palpitations
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 3 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Hepatobiliary disorders
Biliary dilatation
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Hepatobiliary disorders
Gallstones
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Injury, poisoning and procedural complications
Bruising
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Injury, poisoning and procedural complications
Knee injury
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Ear and labyrinth disorders
Car sickness
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 2 • up to 30 days after the last dose
|
|
Immune system disorders
Allergy
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Reproductive system and breast disorders
Menstrual cramps
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Surgical and medical procedures
Hospitalization
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Vomiting
|
6.0%
3/50 • Number of events 3 • up to 30 days after the last dose
|
13.7%
7/51 • Number of events 13 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 2 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Nausea
|
4.0%
2/50 • Number of events 2 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
6.1%
3/49 • Number of events 3 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Cyclic Vomiting Syndrome
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
3.9%
2/51 • Number of events 2 • up to 30 days after the last dose
|
4.1%
2/49 • Number of events 3 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Stomach pain
|
0.00%
0/50 • up to 30 days after the last dose
|
5.9%
3/51 • Number of events 5 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/50 • up to 30 days after the last dose
|
3.9%
2/51 • Number of events 2 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Abdominal tenderness
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Bloated Feeling
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Erosive esophagitis
|
0.00%
0/50 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
2.0%
1/49 • Number of events 1 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Gas pain
|
0.00%
0/50 • up to 30 days after the last dose
|
2.0%
1/51 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
|
Gastrointestinal disorders
Gastroesohageal reflux disease
|
2.0%
1/50 • Number of events 1 • up to 30 days after the last dose
|
0.00%
0/51 • up to 30 days after the last dose
|
0.00%
0/49 • up to 30 days after the last dose
|
Additional Information
Dan Myers, PhD, VP Product & R&D
Alexza Pharmaceuticals Inc.
Phone: (650) 210-6164
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Alexza Pharmaceuticals has full ownership of the data collected as part of the study. The results of this study may be published in a medical publication, journal, or another public dissemination, presented at a medical conference or used for teaching purposes. This study and its results may be submitted for inclusion in all appropriate health authority study registries, as well as publication on health authority study registry websites, as required by local health authority regulations.
- Publication restrictions are in place
Restriction type: OTHER