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

Phase 2

Completed

Conditions: Cyclic Vomiting Syndrome

Registration Number: NCT04645953

Lead Sponsor: Alexza Pharmaceuticals, Inc.

Brief 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.

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

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 150

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.	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.

Secondary Outcome Measures

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

Trial Locations

Locations (18): Om Research
🇺🇸
Lancaster, California, United States
Axis Clinical Trials
🇺🇸
Los Angeles, California, United States
Precision Research Institute, LLC
🇺🇸
San Diego, California, United States
University of South Florida
🇺🇸
Tampa, Florida, United States
Summit Clinical Studies
🇺🇸
Athens, Georgia, United States
Infinite Clinical Trials
🇺🇸
Morrow, Georgia, United States
Kansas University Medical Center
🇺🇸
Kansas City, Kansas, United States
Massachusetts General Hospital
🇺🇸
Boston, Massachusetts, United States
Henry Ford Health System
🇺🇸
Detroit, Michigan, United States
Mayo Clinic
🇺🇸
Rochester, Minnesota, United States
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Om Research
🇺🇸Lancaster, California, United States