Phase 2
N=150
Staccato Granisetron® (AZ 010) for the Treatment of Cyclic Vomiting Syndrome
Cyclic Vomiting Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04645953 ↗Enrolled (actual)
150
Serious AEs
6.0%
Results posted
Jun 2025
Primary outcome: Primary: The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period. — 0.69; 1.43; 1.33; 1.48 event — p=0.7024
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 3mg AZ-010 (Combination_product); 1mg AZ010 (Combination_product); Staccato Placebo (Combination_product)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexza Pharmaceuticals, Inc.
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period. |
0.69; 1.43; 1.33; 1.48; 2.43; 2.13 | 0.7024 |
| SECONDARY Anxiety/Panic Visual Analog Scale (VAS) Score |
51.30; 52.08; 47.72; 48.25; 64.00; 51.29 | 0.0504 |
| SECONDARY Prior Episode Questionnaire |
1.46; 1.27; 1.52; 1.58; 1.55; 1.43 | 0.8299 |
| SECONDARY Rescue Medication Use Within 1 Day of Dose |
3; 2; 1; 9; 11; 6 | 0.3847 |
| SECONDARY Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office |
2; 0; 0; 45; 49; 44 | 0.9732 |
| SECONDARY Rhodes Index of Nausea, Vomiting, and Retching (RINVR) |
11.20; 11.85; 11.79; 6.57; 10.45; 7.81 | 0.1337 |
| SECONDARY Abdominal Pain, Visual Analog Scale (VAS) Score |
45.48; 53.04; 51.60; 51.50; 47.25; 32.57 | 0.0504 |
| SECONDARY Intensity of Vomiting/Retching Attack |
2.29; 3.00; 2.25; 1.86; 2.29; 1.57 | 0.9664 |
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.
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT04645953). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.