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

Alexza Pharmaceuticals, Inc.150 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

150

Conditions

Cyclic Vomiting Syndrome

Interventions

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)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 YearsHealthy volunteers:

Medical Language ↔ Plain English

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.

Locations (18)

Om Research

Lancaster, California, United States

Axis Clinical Trials

Los Angeles, California, United States

Outcomes

Primary Outcomes

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.

Secondary Outcomes

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)

Data from ClinicalTrials.gov

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