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

NCT04645953 | Completed Phase 2 Results FDA Drug FDA Device

• 1 other identifier: AMDC 010-201
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 18

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.

Conditions

Cyclic Vomiting Syndrome

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

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (7)

• Anxiety/Panic Visual Analog Scale (VAS) Score

  24 hours after treatment dose

• Prior Episode Questionnaire

  24 hours after each dose

• Rescue Medication Use Within 1 Day of Dose

  24 hours post dose

• Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office

  Within Day 1 after dosing

• Rhodes Index of Nausea, Vomiting, and Retching (RINVR)

  Within 24 hours following treatment.

Study Arms (3)

1mg AZ010

EXPERIMENTAL

Single orally-inhaled dose

Combination Product: 1mg AZ010

3mg AZ010

EXPERIMENTAL

Single orally-inhaled dose

Combination Product: 3mg AZ-010

Placebo

EXPERIMENTAL

Single orally-inhaled dose

Combination Product: Staccato Placebo

Interventions

3mg AZ-010 COMBINATION_PRODUCT

Subjects who received a single inhaled dose (3mg)

3mg AZ010

1mg AZ010 COMBINATION_PRODUCT

Subjects who received a single inhaled dose (1mg)

1mg AZ010

Staccato Placebo COMBINATION_PRODUCT

Subject who received a single inhaled dose (Staccato Placebo)

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexza Pharmaceuticals, Inc.: lead

Study Sites (18)

Om Research

Lancaster, California, 93535, United States

Location

Axis Clinical Trials

Los Angeles, California, 90036, United States

Location

Precision Research Institute, LLC

San Diego, California, 92114, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

Summit Clinical Studies

Athens, Georgia, 30607, United States

Location

Infinite Clinical Trials

Morrow, Georgia, 30260, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66160, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

NY Scientific

Brooklyn, New York, 11235, United States

Location

Temple University Hospital

Philadelphia, Pennsylvania, 19140, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

New Phase Research & Development, LLC

Knoxville, Tennessee, 37909, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Pioneer Research Solutions

Houston, Texas, 77099, United States

Location

Sante Clinical Research

Kerrville, Texas, 78028, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Familial cyclic vomiting syndrome; Vomiting; Migraine Disorders; Nausea; Abdominal Pain; Foodborne Diseases

Condition Hierarchy (Ancestors)

Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Headache Disorders, Primary; Headache Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Pain; Neurologic Manifestations; Poisoning; Chemically-Induced Disorders

Limitations and Caveats

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.

Results Point of Contact

• Title: Dan Myers, PhD, VP Product & R&D
• Organization: Alexza Pharmaceuticals Inc.

dmyers@alexza.com

(650) 210-6164

Study Officials

• Larry Carter, PhD

  Alexza Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2020
• First Posted: November 27, 2020
• Study Start: February 5, 2021
• Primary Completion: July 26, 2022
• Study Completion: July 26, 2022
• Last Updated: June 11, 2025
• Results First Posted: June 11, 2025

Record last verified: 2025-03

Locations

US (18)