Cyclic Vomiting SyndromePhase 2NCT04645953

Staccato Granisetron® (AZ 010) for the Treatment of Cyclic Vomiting Syndrome

A Phase 2 study testing 3mg AZ-010 and 1mg AZ010 in people with Cyclic Vomiting Syndrome, run by Alexza Pharmaceuticals, Inc. at 18 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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. Read more about this study

Who is it for?: Ages 18 to 60 yearsHealthy volunteers can take part
Conditions the study is for: Cyclic Vomiting Syndrome
What is being tested?: 3mg AZ-010 and 1mg AZ010
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 150 participants
Where: 18 sitesUnited States
Study dates: 5 Feb 2021to 26 Jul 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 60 years
All sexes
Healthy volunteers accepted
Adult

Who may be able to join

Adult males and females between 18 and 60 years of age, inclusive at the time of signing the informed consent document.
Diagnosis of cyclic vomiting syndrome (CVS) using the Rome IV diagnostic criteria.
Otherwise healthy, as determined by the responsible physician, based on a medical evaluation including history, physical examination, vital signs, electrocardiograms (ECGs) and laboratory tests assessed at the screening visit
Negative urine tests for selected drugs of abuse and alcohol breath test at Screening.

Who cannot join

Any significant medical or psychiatric condition that could, in the Investigator's opinion, compromise the subject's safety or interfere with the completion of this protocol.
Any condition, including the presence of laboratory abnormalities or pulmonary condition, which according to the Investigator places the subject at unacceptable risk if he/she were to participate in the study.
A diagnosis of any gastrointestinal disorder other than CVS that in the judgement of the Investigator could compromise the subject's safety or interfere with the interpretation of safety or efficacy data.

Trial locations and contacts

18 sites in 1 country.

City, postcode or hospital

18 sites in 1 country

United States18 sites

Summit Clinical Studies
Athens, Georgia, 30607
Massachusetts General Hospital
Boston, Massachusetts, 02114
NY Scientific
Brooklyn, New York, 11235
Henry Ford Health System
Detroit, Michigan, 48202
Baylor College of Medicine
Houston, Texas, 77030
Pioneer Research Solutions
Houston, Texas, 77099
Kansas University Medical Center
Kansas City, Kansas, 66160
Sante Clinical Research
Kerrville, Texas, 78028
New Phase Research & Development, LLC
Knoxville, Tennessee, 37909
Om Research
Lancaster, California, 93535
Axis Clinical Trials
Los Angeles, California, 90036
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Infinite Clinical Trials
Morrow, Georgia, 30260
Temple University Hospital
Philadelphia, Pennsylvania, 19140
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Mayo Clinic
Rochester, Minnesota, 55905
Precision Research Institute, LLC
San Diego, California, 92114
University of South Florida
Tampa, Florida, 33606

Other trials in United States

About this study

Official title: 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

Read the detailed description

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

Conditions: Cyclic Vomiting Syndrome
Being tested: 3mg AZ-010, 1mg AZ010, Staccato Placebo
Keywords: Cyclic Vomiting Syndrome, CVS, Vomiting, Retching, Abdominal migraine, Nausea, Stomach pain, Unexplained vomiting, Functional GI Disorder, CVSHOPE.com, Throwing up, Puking, Dry heaving, Food poisoning, Episodes, Repeated food poisoning
Medical subject headings: Familial cyclic vomiting syndrome, Vomiting, Migraine Disorders, Nausea, Abdominal Pain, Foodborne Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

3mg AZ-010Combination product
Subjects who received a single inhaled dose (3mg)
1mg AZ010Combination product
Subjects who received a single inhaled dose (1mg)
Staccato PlaceboCombination product
Subject who received a single inhaled dose (Staccato Placebo)

Study groups in NCT04645953
Study group	Role	What they receive
1mg AZ010	Experimental	Single orally-inhaled dose Receives: Combination Product: 1mg AZ010
3mg AZ010	Experimental	Single orally-inhaled dose Receives: Combination Product: 3mg AZ-010
Placebo	Experimental	Single orally-inhaled dose Receives: Combination Product: Staccato Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.

Secondary outcomes (7)

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.
Measured: 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)
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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).
Measured: Within 24 hours post dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

5 Feb 202126 Jul 2022

First posted27 Nov 2020
Study start5 Feb 2021
Primary completion26 Jul 2022
Study completion26 Jul 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cyclic Vomiting SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Investigator, Outcomes assessor
Enrolment: 150 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04645953
Sponsor's protocol ID: AMDC 010-201
Status last verified: Mar 2025
First submitted: 16 Nov 2020
Last updated: 11 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cyclic Vomiting Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT04645953
Acronym / sponsor ID: AMDC 010-201
Conditions: Cyclic Vomiting Syndrome
Keywords: Cyclic Vomiting Syndrome, CVS, Vomiting, Retching, Abdominal migraine, Nausea, Stomach pain, Unexplained vomiting
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 150 participants (actual)
Lead sponsor: Alexza Pharmaceuticals, Inc.
Study start: 5 Feb 2021 (actual)
Primary completion: 26 Jul 2022 (actual)
Study completion: 26 Jul 2022 (actual)
First posted: 27 Nov 2020 (actual)
Results: Posted 11 Jun 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
1mg AZ010	Experimental	Combination Product: 1mg AZ010Single orally-inhaled dose
3mg AZ010	Experimental	Combination Product: 3mg AZ-010Single orally-inhaled dose
Placebo	Experimental	Combination Product: Staccato PlaceboSingle orally-inhaled dose

Who may qualify

Ages: 18 to 60 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult

Key inclusion criteria (4 listed)

Adult males and females between 18 and 60 years of age, inclusive at the time of signing the informed consent document.
Diagnosis of cyclic vomiting syndrome (CVS) using the Rome IV diagnostic criteria.
Otherwise healthy, as determined by the responsible physician, based on a medical evaluation including history, physical examination, vital signs, electrocardiograms (ECGs) and laboratory tests assessed at the screening visit
Negative urine tests for selected drugs of abuse and alcohol breath test at Screening.

Key exclusion criteria (3 listed)

Any significant medical or psychiatric condition that could, in the Investigator's opinion, compromise the subject's safety or interfere with the completion of this protocol.
Any condition, including the presence of laboratory abnormalities or pulmonary condition, which according to the Investigator places the subject at unacceptable risk if he/she were to participate in the study.
A diagnosis of any gastrointestinal disorder other than CVS that in the judgement of the Investigator could compromise the subject's safety or interfere with the interpretation of safety or efficacy data.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
3mg AZ-010Subjects who received a single inhaled dose (3mg)	Combination product	3mg AZ010
1mg AZ010Subjects who received a single inhaled dose (1mg)	Combination product	1mg AZ010
Staccato PlaceboSubject who received a single inhaled dose (Staccato Placebo)	Combination product	Placebo

Primary outcome

The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
Time frame: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.
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.

7 secondary outcomes

Anxiety/Panic Visual Analog Scale (VAS) Score
Time frame: 24 hours after treatment dose
Prior Episode Questionnaire
Time frame: 24 hours after each dose
Rescue Medication Use Within 1 Day of Dose
Time frame: 24 hours post dose
Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office
Time frame: Within Day 1 after dosing
Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
Time frame: Within 24 hours following treatment.
Abdominal Pain, Visual Analog Scale (VAS) Score
Time frame: Up to 24 hours post dose.
Intensity of Vomiting/Retching Attack
Time frame: Within 24 hours post dose.

Results and safety

Results posted to the registry on 11 Jun 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
1mg AZ010	50	48
3mg AZ010	51	48
Placebo	49	46

Primary outcome: The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.

Time frame: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.. Mean (Standard Deviation). Unit: event

Measure	1mg AZ010 n = 47	3mg AZ010 n = 49	Placebo n = 48
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)

1mg AZ010: p = 0.7024, ANOVA
3mg AZ010: p = 0.2051, ANOVA

Population analysed: 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.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: up to 30 days after the last dose. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
1mg AZ010	3 of 50 (6%)	28 of 50 (56%)	0 of 50
3mg AZ010	3 of 51 (5.9%)	40 of 51 (78.4%)	0 of 51
Placebo	3 of 49 (6.1%)	44 of 49 (89.8%)	0 of 49

Limitations and caveats (from the sponsor): The complexity of data collection interface/instructions and patient impairment while experiencing severe CVS episodes may likely have led to the high incidence of missing data with impaired patients missing data collection timepoints. A simplified data collection interface, clear instructions, and potentially collection of fewer overall parameters may make it easier for patients in future studies to record complete study data.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cyclic Vomiting Syndrome

Enrolling by invitationObservational study
Body Surface Gastric Mapping to Evaluate Patients With Upper Gastrointestinal Symptoms and Controls
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Recruiting
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Cyclic Vomiting Syndrome
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A Study of Auricular Neurostimulation for Children With Cyclic Vomiting Syndrome
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Recruiting
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Larry Carter, PhD study director

Alexza Pharmaceuticals, Inc.

18 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing 3mg AZ-010 and 1mg AZ010 in people with Cyclic Vomiting Syndrome, run by Alexza Pharmaceuticals, Inc. at 18 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period. (time frame: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04645953.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2025.