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

• ClinicalTrials.gov Identifier: NCT04645953
• Recruitment Status: Completed
• First Posted: November 27, 2020
• Last Update Posted: September 27, 2022
• Sponsor: Alexza Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Alexza Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: November 16, 2020
• First Posted Date: November 27, 2020
• Last Update Posted Date: September 27, 2022
• Actual Study Start Date: February 11, 2021
• Actual Primary Completion Date: July 19, 2022 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 25, 2020): The number of vomiting/retching events in the 2 hours following treatment. [ Time Frame: 24 hours ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: November 25, 2020): Number of adverse events as a measure of safety [ Time Frame: 24 hours ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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
• Study Type: Interventional
• Study Phase: Phase 2

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description:

Double Blind

Primary Purpose: Treatment

• Condition: Cyclic Vomiting Syndrome

Intervention

• Combination Product: 3mg AZ-010
  Subject will receive a single inhaled dose (3mg)
• Combination Product: 1mg AZ010
  Subject will receive a single inhaled dose (1mg)
• Combination Product: Staccato Placebo
  Subject will receive a single inhaled dose (Staccato Placebo)

Study Arms

• Experimental: 1mg AZ010
  Single orally-inhaled dose
  Intervention: Combination Product: 1mg AZ010
• Experimental: 3mg AZ010
  Single orally-inhaled dose
  Intervention: Combination Product: 3mg AZ-010
• Experimental: Placebo
  Single orally-inhaled dose
  Intervention: Combination Product: Staccato Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 26, 2022): 151
• Original Estimated Enrollment (submitted: November 25, 2020): 150
• Actual Study Completion Date: July 19, 2022
• Actual Primary Completion Date: July 19, 2022 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 60 Years (Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04645953
• Other Study ID Numbers: AMDC 010-201
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Alexza Pharmaceuticals, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Alexza Pharmaceuticals, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Larry Carter, PhD; Alexza Pharmaceuticals, Inc.

• PRS Account: Alexza Pharmaceuticals, Inc.
• Verification Date: September 2022