Clinical trial NCT04645953: Staccato Granisetron® (AZ 010) for the Treatment of Cyclic Vomiting Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: Alexza Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2020-11-27
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

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.

Primary outcomes:
measure: The Number of Vomiting/Retching Events Reported by Study Participants Following Treatment/Dosing During the Home Treament Period.
timeFrame: Within the following timepoints: 30, 60, 90 and 120-minutes post-dose.
Secondary outcomes:
measure: Anxiety/Panic Visual Analog Scale (VAS) Score
timeFrame: 24 hours after treatment dose
measure: Prior Episode Questionnaire
timeFrame: 24 hours after each dose
measure: Rescue Medication Use Within 1 Day of Dose
timeFrame: 24 hours post dose
measure: Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office
timeFrame: Within Day 1 after dosing
measure: Rhodes Index of Nausea, Vomiting, and Retching (RINVR)
timeFrame: Within 24 hours following treatment.
measure: Abdominal Pain, Visual Analog Scale (VAS) Score
timeFrame: Up to 24 hours post dose.
measure: Intensity of Vomiting/Retching Attack
timeFrame: Within 24 hours post dose.
Other outcomes:
Locations:
city: Lancaster
country: United States
facility: Om Research
state: California
city: Los Angeles
country: United States
facility: Axis Clinical Trials
state: California
city: San Diego
country: United States
facility: Precision Research Institute, LLC
state: California
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Athens
country: United States
facility: Summit Clinical Studies
state: Georgia
city: Morrow
country: United States
facility: Infinite Clinical Trials
state: Georgia
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Brooklyn
country: United States
facility: NY Scientific
state: New York
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Knoxville
country: United States
facility: New Phase Research & Development, LLC
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Pioneer Research Solutions
state: Texas
city: Kerrville
country: United States
facility: Sante Clinical Research
state: Texas
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Alexza Pharmaceuticals, Inc.
name: Larry Carter, PhD
role: STUDY_DIRECTOR
References: