Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1mg AZ010 | Experimental | Single orally-inhaled dose |
|
| 3mg AZ010 | Experimental | Single orally-inhaled dose |
|
| Placebo | Experimental | Single orally-inhaled dose |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 3mg AZ-010 | Combination Product | Subjects who received a single inhaled dose (3mg) |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Larry Carter, PhD | Alexza Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Om Research | Lancaster | California | 93535 | United States | ||
| Axis Clinical Trials |
Participant eligibility inclusion and exclusion criteria via the following assessments: Medical history (with particular reference to history of vomiting symptoms, CVS diagnosis, typical duration of CVS episode, average # of vomiting/retching events in typical CVS episode, frequency of CVS episodes per year, any triggers for CVS episode, conditions that may cause similar symptoms, treatment history, and current medications, medical history, COVID-19 screening.
This study was a multicenter study conducted at 18 sites in the United States. Of these, only 17 sites were activated, and only 15 sites enrolled subjects.
Study Period: 1 year 5 months Study Start: 05 February 2021 Study End: 26 July 2022 50 patients randomized to each of 3 treatment groups: 1 mg AZ-010, 3 mg AZ-010, or matching Staccato® Placebo Patients were screened at the Research Clinic to determine eligibility for study participation no more than14 days prior to Visit 2.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 1mg AZ010 | Single orally-inhaled dose 1mg AZ010: Subject will receive a single inhaled dose (1mg) single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. |
| FG001 | 3mg AZ010 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 3, 2022 |
Not provided
Not provided
Not provided
Not provided
Not provided
Double Blind
| 1mg AZ010 |
| Combination Product |
Subjects who received a single inhaled dose (1mg) |
|
| Staccato Placebo | Combination Product | Subject who received a single inhaled dose (Staccato Placebo) |
|
| 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) | 24 hours after each dose |
| Rescue Medication Use Within 1 Day of 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. | 24 hours post dose |
| Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office | 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. | Within Day 1 after dosing |
| Rhodes Index of Nausea, Vomiting, and Retching (RINVR) | 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. | Within 24 hours following treatment. |
| Abdominal Pain, Visual Analog Scale (VAS) Score | 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 | Up to 24 hours post dose. |
| Intensity of Vomiting/Retching Attack | 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). | Within 24 hours post dose. |
| Los Angeles |
| California |
| 90036 |
| United States |
| Precision Research Institute, LLC | San Diego | California | 92114 | United States |
| University of South Florida | Tampa | Florida | 33606 | United States |
| Summit Clinical Studies | Athens | Georgia | 30607 | United States |
| Infinite Clinical Trials | Morrow | Georgia | 30260 | United States |
| Kansas University Medical Center | Kansas City | Kansas | 66160 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| NY Scientific | Brooklyn | New York | 11235 | United States |
| Temple University Hospital | Philadelphia | Pennsylvania | 19140 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| New Phase Research & Development, LLC | Knoxville | Tennessee | 37909 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Pioneer Research Solutions | Houston | Texas | 77099 | United States |
| Sante Clinical Research | Kerrville | Texas | 78028 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
Single orally-inhaled dose
3mg AZ-010: Subject will receive a single inhaled dose (3mg)
(with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date.
| FG002 | Placebo | Single orally-inhaled dose Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 1mg AZ010 | Single orally-inhaled dose 1mg AZ010: Subject will receive a single inhaled dose (1mg) |
| BG001 | 3mg AZ010 | Single orally-inhaled dose 3mg AZ-010: Subject will receive a single inhaled dose (3mg) |
| BG002 | Placebo | Single orally-inhaled dose Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo) |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Height | Mean | Standard Deviation | cm |
| |||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||
| Body Mass Index (kg/m2) | Mean | Standard Deviation | kg/m^2 |
| |||||||||||||||
| Childbearing potential (Yes) | Count of Participants | Participants |
| ||||||||||||||||
| Childbearing potential (No) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Mean | Standard Deviation | event | Within the following timepoints: 30, 60, 90 and 120-minutes post-dose. |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Mean | Standard Deviation | score on a scale | 24 hours after treatment dose |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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) | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Mean | Standard Deviation | score on a scale | 24 hours after each dose |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Rescue Medication Use Within 1 Day of 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. | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Count of Participants | Participants | 24 hours post dose |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Health Care Provider Visits; Visit to Urgent Care, Emergency Department, or Physician's Office | 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. | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Count of Participants | Participants | Within Day 1 after dosing |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Rhodes Index of Nausea, Vomiting, and Retching (RINVR) | 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. | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Mean | Standard Deviation | score on a scale | Within 24 hours following treatment. |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Abdominal Pain, Visual Analog Scale (VAS) Score | 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 | Per protocol population (CVS patients between 18 and 60 years of age who experienced at least 3 episodes of recurrent vomiting in the last 12 months and 2 episodes in the last 6 months. | Posted | Mean | Standard Deviation | units on a scale | Up to 24 hours post dose. |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Intensity of Vomiting/Retching Attack | 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). | Per protocol population (CVS patients between 18 and 60 years of age who reported intensity of vomiting attack. | Posted | Mean | Standard Deviation | units on a scale | Within 24 hours post dose. |
|
up to 30 days after the last dose
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 1mg AZ010 | Single orally-inhaled dose 1mg AZ010: Subject will receive a single inhaled dose (1mg) single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. | 0 | 50 | 3 | 50 | 28 | 50 |
| EG001 | 3mg AZ010 | Single orally-inhaled dose 3mg AZ-010: Subject will receive a single inhaled dose (3mg) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. | 0 | 51 | 3 | 51 | 40 | 51 |
| EG002 | Placebo | Single orally-inhaled dose Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. | 0 | 49 | 3 | 49 | 44 | 49 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| CVS, Nausea | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment |
| |
| Surgical and medical procedures | Surgical and medical procedures | MedDRA, version 23.1 | Systematic Assessment |
| |
| Biliary dilatation | Hepatobiliary disorders | MedDRA, version 23.1 | Systematic Assessment | 3 mg AZ-010 |
|
| Hypertension | Vascular disorders | MedDRA, version 23.1 | Systematic Assessment | Placebo |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg, placebo AZ010 |
|
| Nausea | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg, placebo AZ010 |
|
| Cyclic Vomiting Syndrome | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg, placebo AZ010 |
|
| Stomach pain | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg, placebo AZ010 |
|
| Constipation | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Abdominal tenderness | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Bloated Feeling | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Erosive esophagitis | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Gas pain | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Gastroesohageal reflux disease | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Hematemesis | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Indigestion | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Pain abdominal | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Retching | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Vomited | Gastrointestinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Headache | Nervous system disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, placebo AZ010 |
|
| Burning sensation | Nervous system disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Dizziness | Nervous system disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Migraine | Nervous system disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| COVID-19 | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg, placebo AZ010 |
|
| Acute diverticulitis | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Acute pharyngitis | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Acute sinusitis | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Rhinovirus infection | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Yeast infection | Infections and infestations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Hypokalemia | Metabolism and nutrition disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg placebo AZ010 |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg, placebo AZ010 |
|
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Hypophosphatemia | Metabolism and nutrition disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Metabolic alkalosis | Metabolism and nutrition disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Anxiety | Psychiatric disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Panic attack | Psychiatric disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, placebo AZ010 |
|
| Depersonalization | Psychiatric disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Difficulty sleeping | Psychiatric disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Stress | Psychiatric disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, 3mg AZ010 |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg, placebo AZ010 |
|
| Dry cough | Respiratory, thoracic and mediastinal disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Nosebleed | Respiratory, thoracic and mediastinal disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Fatigue | General disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg, placebo AZ010 |
|
| Fever | General disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Pain | General disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Poikilothermia | General disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Tiredness | General disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Generalized muscle aches | Musculoskeletal and connective tissue disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg, placebo AZ010 |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Muscle cramps | Musculoskeletal and connective tissue disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Weakness in extremity | Musculoskeletal and connective tissue disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Flushing | Vascular disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Hypertension | Vascular disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Hypotension | Vascular disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Pale skin | Vascular disorders | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Endoscopic retrograde cholangiopancreatography | Investigations | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Lactate increased | Investigations | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| QT interval prolonged | Investigations | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| ST segment depressed | Investigations | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
| Transaminitis | Investigations | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Redness | Skin and subcutaneous tissue disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Sweating | Skin and subcutaneous tissue disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Left anterior fascicular block | Cardiac disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Palpitations | Cardiac disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Biliary dilatation | Hepatobiliary disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Gallstones | Hepatobiliary disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Bruising | Injury, poisoning and procedural complications | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Knee injury | Injury, poisoning and procedural complications | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Car sickness | Ear and labyrinth disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Allergy | Immune system disorders | MedDRA, version 23.1 | Systematic Assessment | 3mg AZ010 |
|
| Menstrual cramps | Reproductive system and breast disorders | MedDRA, version 23.1 | Systematic Assessment | placebo AZ010 |
|
| Hospitalization | Surgical and medical procedures | MedDRA, version 23.1 | Systematic Assessment | 1mg AZ010 |
|
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.
Alexza Pharmaceuticals has full ownership of the data collected as part of the study. The results of this study may be published in a medical publication, journal, or another public dissemination, presented at a medical conference or used for teaching purposes. This study and its results may be submitted for inclusion in all appropriate health authority study registries, as well as publication on health authority study registry websites, as required by local health authority regulations.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dan Myers, PhD, VP Product & R&D | Alexza Pharmaceuticals Inc. | (650) 210-6164 | dmyers@alexza.com |
| Feb 5, 2025 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| C536228 | Familial cyclic vomiting syndrome |
| D014839 | Vomiting |
| D008881 | Migraine Disorders |
| D009325 | Nausea |
| D015746 | Abdominal Pain |
| D005517 | Foodborne Diseases |
| ID | Term |
|---|---|
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D051270 | Headache Disorders, Primary |
| D020773 | Headache Disorders |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D010146 | Pain |
| D009461 | Neurologic Manifestations |
| D011041 | Poisoning |
| D064419 | Chemically-Induced Disorders |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Number Vomiting & Retching Events First Available Efficacy Data, 1 hour post dose |
|
|
| Number Vomiting & Retching Events First Available Efficacy Data, 90 minutes post dose |
|
|
| Number Vomiting & Retching Events First Available Efficacy Data, 2 hours post dose |
|
|
| ANOVA |
| 0.2051 |
| Superiority |
Null hypothesis is there was no difference between groups treated with AZ-010 (1 mg or 3 mg) and the placebo group in the mean number of vomiting/retching events in the 2 hours following. treatment. Baseline, body weight, height, body mass index, age as covariates, and the treatment group and study site as factors. Patients summarized by actual treatment received. Formal statistical tests (ANOVA, when performed) were 2-sided t-tests with a significance value of 0.05. |
|
|
|
| OG002 | Placebo | Single orally-inhaled dose Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
Single orally-inhaled dose 3mg AZ-010: Subject will receive a single inhaled dose (3mg) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. |
| OG002 | Placebo | Single orally-inhaled dose Staccato Placebo: Subject will receive a single inhaled dose (Staccato Placebo) (with single daily dosing up to 5 consecutive days) within 12 weeks after the randomization date. |
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|