A Phase 2 interventional study of 3mg AZ-010 and 1mg AZ010 in Cyclic Vomiting Syndrome, sponsored by Alexza Pharmaceuticals, Inc.. Completed at 18 sites in United States. Open to participants aged 18 Years to 60 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-06-11.
Sponsored by Alexza Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
This is a multicenter, randomized, double-blind, parallel group, placebo-controlled, efficacy and safety study of adult outpatients diagnosed with CVS and experiencing recurring episodes of stereotypical vomiting.
A Randomized, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of Staccato Granisetron (AZ-010) for the Acute Treatment of Moderate to Severe Cyclic Vomiting Syndrome
9,217 studies on the registry are indexed under Syndrome; 1,031 are open to participants now.
This study's enrollment of 150 is above the median of 50 across 6,515 interventional studies indexed under Syndrome.
Browse Syndrome studies →Alexza Pharmaceuticals, Inc. is the lead sponsor of 25 studies on the registry; none are open to participants now.
Of its 23 completed or terminated interventional studies of FDA-regulated products, 13 (57%) have results posted.
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Exclusion Criteria:
Single orally-inhaled dose
Combination Product: 1mg AZ010
Single orally-inhaled dose
Combination Product: 3mg AZ-010
Single orally-inhaled dose
Combination Product: Staccato Placebo
Subjects who received a single inhaled dose (3mg)
Subjects who received a single inhaled dose (1mg)
Subject who received a single inhaled dose (Staccato Placebo)
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
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.
Time frame: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.
Anxiety/Panic Visual Analog Scale (VAS) Score
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.
Time frame: 24 hours after treatment dose
Prior Episode Questionnaire
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)
Time frame: 24 hours after each dose
Rescue Medication Use Within 1 Day of Dose
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.
Time frame: 24 hours post dose
Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office
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.
Time frame: Within Day 1 after dosing
Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
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.
Time frame: Within 24 hours following treatment.
Abdominal Pain, Visual Analog Scale (VAS) Score
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
Time frame: Up to 24 hours post dose.
Intensity of Vomiting/Retching 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).
Time frame: Within 24 hours post dose.
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.
| Milestone | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Started | 50 | 51 | 49 |
| Completed | 48 | 48 | 46 |
| Not completed | 2 | 3 | 3 |
| Withdrew: Lost to follow-up | 2 | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 | 0 |
| Withdrew: Physician decision | 0 | 1 | 0 |
| Withdrew: Discontinued prior to dosing | 0 | 0 | 2 |
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.
| event | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Number Vomiting & Retching Events, First Available Efficacy Data, 30 minutes post dose | 0.69 ± 1.168 | 1.43 ± 1.975 | 1.33 ± 2.239 |
| Number Vomiting & Retching Events First Available Efficacy Data, 1 hour post dose | 1.48 ± 2.694 | 2.43 ± 3.099 | 2.13 ± 3.180 |
| Number Vomiting & Retching Events First Available Efficacy Data, 90 minutes post dose | 2.17 ± 4.243 | 3.32 ± 3.906 | 2.59 ± 4.205 |
| Number Vomiting & Retching Events First Available Efficacy Data, 2 hours post dose | 3.10 ± 5.576 | 4.21 ± 5.279 | 2.61 ± 4.408 |
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.
| score on a scale | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Anxiety/Panic VAS Score, 2 hour, post-dose | 51.30 ± 34.907 | 52.08 ± 35.020 | 47.72 ± 32.849 |
| Anxiety/Panic VAS Score, 6 hour, post-dose | 48.25 ± 17.343 | 64.00 ± 35.071 | 51.29 ± 20.605 |
| Anxiety/Panic VAS Score, 12 hour, post-dose | 31.00 ± 28.827 | 51.15 ± 31.947 | 64.00 ± 26.533 |
| Anxiety/Panic VAS Score, 24 hour, post-dose | 18.00 ± 19.378 | 37.88 ± 31.439 | 62.25 ± 30.478 |
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)
| score on a scale | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Prior Episode Questionnaire, 24 Hours Post-Dose, Duration Of Current Episode | 1.46 ± 0.658 | 1.27 ± 0.550 | 1.52 ± 0.680 |
| Prior Episode Questionnaire, 24 Hours Post-Dose, Intensity Of Current Episode | 1.58 ± 0.654 | 1.55 ± 0.671 | 1.43 ± 0.598 |
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.
| Participants | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Percentage of patients confirming "Yes" response to rescue medication use, 2 hours post dose | 3 | 2 | 1 |
| Percentage of patients confirming "Yes" response to rescue medication use, 24 hours post dose | 9 | 11 | 6 |
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.
| Participants | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Patients requiring healthcare provider visit within 24 hours post dose of CVS episode. | 2 | 0 | 0 |
| Patients not requiring healthcare provider visit within 24 hours post dose of CVS episode. | 45 | 49 | 44 |
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.
| score on a scale | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 6 hour, post-dose | 11.20 ± 7.741 | 11.85 ± 7.893 | 11.79 ± 7.695 |
| Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 12 hour, post-dose | 6.57 ± 7.710 | 10.45 ± 9.237 | 7.81 ± 8.274 |
| Rhodes Index of Nausea, Vomiting and Retching (RINVR) Composite Score, 24 hour, post-dose | 6.76 ± 7.096 | 8.87 ± 8.471 | 7.92 ± 8.057 |
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
| units on a scale | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Abdominal Pain VAS Score,2 hour, post-dose | 45.48 ± 27.318 | 53.04 ± 32.227 | 51.60 ± 29.390 |
| Abdominal Pain VAS Score,6 hour, post-dose | 51.50 ± 17.849 | 47.25 ± 33.970 | 32.57 ± 22.846 |
| Abdominal Pain VAS Score,12 hour, post-dose | 36.67 ± 7.024 | 51.38 ± 33.393 | 61.25 ± 30.489 |
| Abdominal Pain VAS Score, 24 hour, post-dose | 32.60 ± 17.771 | 43.88 ± 38.264 | 66.25 ± 11.354 |
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).
| units on a scale | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| Intensity of Attack Scale (Efficacy Population) 2 hours post-dose | 2.29 ± 0.488 | 3.00 ± 0.894 | 2.25 ± 0.957 |
| Intensity of Attack Scale (Efficacy Population) 6 hour, post-dose | 1.86 ± 0.690 | 2.29 ± 0.756 | 1.57 ± 0.787 |
| Intensity of Attack Scale (Efficacy Population) 12 hour, post-dose | 1.33 ± 0.577 | 2.31 ± 0.947 | 1.75 ± 0.957 |
| Intensity of Attack Scale (Efficacy Population) 24 hour, post-dose | 2.20 ± 0.837 | 2.38 ± 0.916 | 2.25 ± 0.957 |
Collected over up to 30 days after the last dose. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 1mg AZ010 | 0/50 (0%) | 3/50 (6%) | 28/50 (56%) |
| 3mg AZ010 | 0/51 (0%) | 3/51 (5.9%) | 40/51 (78.4%) |
| Placebo | 0/49 (0%) | 3/49 (6.1%) | 44/49 (89.8%) |
| Event | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| CVS, NauseaGastrointestinal disorders | 2/50 | 2/51 | 2/49 |
| HypertensionVascular disorders | 0/50 | 0/51 | 1/49 |
| Surgical and medical proceduresSurgical and medical procedures | 1/50 | 0/51 | 0/49 |
| Biliary dilatationHepatobiliary disorders | 0/50 | 1/51 | 0/49 |
| Event | 1mg AZ010 | 3mg AZ010 | Placebo |
|---|---|---|---|
| VomitingGastrointestinal disorders | 3/50 | 7/51 | 2/49 |
| HeadacheNervous system disorders | 3/50 | 0/51 | 4/49 |
| NauseaGastrointestinal disorders | 2/50 | 1/51 | 3/49 |
| Stomach painGastrointestinal disorders | 0/50 | 3/51 | 1/49 |
| Cyclic Vomiting SyndromeGastrointestinal disorders | 1/50 | 2/51 | 2/49 |
| COVID-19Infections and infestations | 1/50 | 1/51 | 2/49 |
| HypokalemiaMetabolism and nutrition disorders | 1/50 | 1/51 | 2/49 |
| AnxietyPsychiatric disorders | 0/50 | 0/51 | 2/49 |
| ConstipationGastrointestinal disorders | 0/50 | 2/51 | 0/49 |
| Erosive esophagitisGastrointestinal disorders | 0/50 | 0/51 | 1/49 |
| Age, Continuous(years) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Mean | 33.5 ± 11.95 | 36.0 ± 12.30 | 36.2 ± 10.22 | 35.2 ± 11.53 |
| Sex: Female, Male(Participants) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Female | 34 | 34 | 36 | 104 |
| Male | 16 | 17 | 13 | 46 |
| Ethnicity (NIH/OMB)(Participants) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Hispanic or Latino | 8 | 5 | 5 | 18 |
| Not Hispanic or Latino | 41 | 45 | 41 | 127 |
| Unknown or Not Reported | 1 | 1 | 3 | 5 |
| Race (NIH/OMB)(Participants) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 0 | 1 |
| Asian | 1 | 1 | 1 | 3 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 7 | 4 | 3 | 14 |
| White | 41 | 45 | 44 | 130 |
| More than one race | 0 | 1 | 1 | 2 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Height(cm) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Mean | 168.85 ± 9.166 | 168.73 ± 9.729 | 168.81 ± 7.910 | 168.79 ± 8.919 |
| Weight(kg) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Mean | 83.28 ± 24.854 | 78.85 ± 19.500 | 82.08 ± 26.443 | 81.38 ± 23.655 |
| Body Mass Index (kg/m2)(kg/m^2) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Mean | 28.979 ± 7.7236 | 27.529 ± 6.0793 | 28.238 ± 7.9544 | 28.244 ± 7.2635 |
| Childbearing potential (Yes)(Participants) | 1mg AZ010 | 3mg AZ010 | Placebo | Total |
|---|---|---|---|---|
| Count of participants | 24 | 29 | 27 | 80 |
1 further baseline measures are reported on the registry.
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Alexza Pharmaceuticals, Inc.