Prevention of Asthma Exacerbations Using Dupilumab in Urban Children and Adolescents (PANDA)
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|ClinicalTrials.gov Identifier: NCT05347771|
Recruitment Status : Recruiting
First Posted : April 26, 2022
Last Update Posted : June 6, 2022
|Condition or disease||Intervention/treatment||Phase|
|Asthma||Drug: Dupilumab Drug: Placebo||Phase 2|
Protocol CAUSE-01 PANDA is a multicenter, double-blind, placebo-controlled, randomized trial of dupilumab adjunctive therapy for the reduction of asthma exacerbations in urban children and adolescents 6 to 17 years with T2-high exacerbation-prone asthma. Approximately 240 participants will be randomized 2:1 to one of two study arms: 1) guidelines-based asthma treatment + dupilumab, or 2) guidelines-based asthma treatment + placebo. Study treatment will continue for 1 year with an additional 3 months of follow-up following completion of study treatment.
An initial Screening Visit will be followed by a 4-week run-in period. After the run-in, participants who continue to meet eligibility criteria will be randomized to one of the 2 treatment arms and receive their first injection. Over the next 2 weeks, participants will return to the clinic for 3 early treatment response visits. Participants will receive injections of dupilumab or placebo administered subcutaneously every two weeks (Q2W) over 12 months. Each participant will have Evaluation and Management (E&M) visits every 3 months where their asthma and rhinitis will be assessed and adjustments made to their medications based on asthma guidelines.
Participants will be asked to monitor and self-report cold symptoms throughout the treatment period. Participants will be asked to complete up to three paired cold visits. At the time of a cold, participants will be asked to come into the clinic for collection of blood and nasal secretions for associated mechanistic studies. Approximately three days after the clinic visit, participants will complete symptom assessments and will be asked to collect nasal samples at home.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||240 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Prevention of Asthma Exacerbations Using Dupilumab in Urban Children and Adolescents (PANDA) (CAUSE-01)|
|Actual Study Start Date :||May 4, 2022|
|Estimated Primary Completion Date :||December 31, 2024|
|Estimated Study Completion Date :||March 31, 2025|
Participants between 12-17 years of age, will receive an initial dose of 600 mg (two 300 mg injections) followed by 300 mg given every other week (Q2W).
Participants between 6-11 years of age will not complete a loading dose and will receive injections based on their body weight:
Participants with a body weight of 15 kg to less than 30 kg, will receive 100mg Q2W.
Participants with a body weight of 30 kg or more, will receive 200 mg Q2W.
Dupilumab is a recombinant DNA-derived humanized IgG4ĸ monoclonal antibody that selectively binds to anti IL-4R monoclonal antibody (mAb).
Placebo Comparator: Placebo
Participants between 12-17 years of age, will receive an initial dose of placebo (two injections) followed by a placebo injection given every other week (Q2W).
Participants between 6-11 years of age will not receive an initial loading dose of placebo and will receive injections Q2W based on their body weight.
The composition of the placebo for dupilumab is the same as the active study drug without the dupilumab.
Other Name: Placebo for Dupilumab
- Number of asthma exacerbations during the 12-month treatment period [ Time Frame: Week 4 (Treatment initiation) - Week 54 (Completion of treatment) ]Asthma exacerbation defined as a prescription of a course of systemic steroids by a clinician or initiation of a course of systemic steroids by a participant to prevent a serious asthma outcome. If a participant initiates and completes a course of systemic steroids without clinician involvement, this course will be counted only if it meets the following dosage: the course for prednisone, prednisolone, or methylprednisolone will be at least 20 mg daily dose for 3 of 5 consecutive days. The course for dexamethasone will be at least a 10 mg single daily dose. If a corticosteroid burst for the treatment of an asthma exacerbation is prescribed by a non-CAUSE clinician, it will be counted regardless of dose.
- Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) % Predicted [ Time Frame: Week 4 to Week 68 ]
FEV1 is air volume exhaled in 1 second during spirometry. FEV1 percent of predicted value is FEV1 converted to a percentage of normal, based on height, weight, and race.
This measurement will be performed by trained and certified clinical research staff according to American Thoracic Society standards as performed routinely in usual care as part of subspecialist management of asthma.
- Days with symptoms, nights with symptoms, and day and night albuterol use. [ Time Frame: Week 4 to Week 68 ]
Number of days with asthma symptoms as defined by the participant report of daytime asthma symptoms over the preceding 14-day period.
Number of nights with asthma symptoms as defined by participant report of nighttime asthma symptoms over the preceding 14-day period.
Number of days with albuterol use as defined by participant report of days of albuterol (bronchodilator) use over the preceding 14-day period.
Number of nights albuterol use as defined by participant report of nights of albuterol (bronchodilator) use over the preceding 14-day period.
- Asthma control measured by the Asthma Control Questionnaire-5 [ Time Frame: Week 4 to Week 60 ]The Asthma Control Questionnaire-5 is a validated tool that will be used to assess overall asthma control over the last 4 weeks. The Range is 0-6, with higher scores indicating worse asthma control.
- Time to first asthma exacerbation [ Time Frame: Week 4 to Week 68 ]Time between initiation of treatment and first asthma exacerbation or the end of participant follow-up visits.
- Quality of life as measured by the PROMIS Asthma Impact Short Forms (Pediatric or Parent Proxy). [ Time Frame: Week 4 to Week 68 ]As measured by the pediatric patient-reported (ages 8-17) or proxy-reported (ages 6-7) PROMIS Asthma Impact Short Forms.
- Asthma burden as measured by Combined Asthma Severity Index (CASI) [ Time Frame: Week 4 to Week 68 ]The Composite Asthma Severity Index (CASI) is a comprehensive severity scale combining multiple facets of asthma severity: impairment, risk, and treatment. The CASI score ranges from 0 to 20 points, with higher scores indicating higher levels of severity, and includes 5 domains: day symptoms and albuterol use, night symptoms and albuterol use, controller treatment, lung function measures, and exacerbations.
- Rhinitis symptoms as measured by Modified Rhinitis Symptoms Utility Index (MRSUI) [ Time Frame: Week 4 - Week 68 ]The Modified Rhinitis Symptom Utility Index (MRSUI), assesses the frequency and severity (degree of bothering: not bothered, somewhat bothered, bothered a lot) of the participant's (1) stuffy or blocked nose, (2) runny nose, (3) sneezing, (4) itchy, watery eyes, and (5) itchy nose or throat over the preceding 14-day period.
- Related adverse events and serious adverse events in the course of treatment [ Time Frame: Week 4 to Week 68 ]
The number of adverse events (AEs) by severity and relationship to study drug will be used to assess safety.
The number of serious adverse events (SAEs) by severity and relationship to study drug will be used to assess safety.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05347771
|United States, Colorado|
|Children's Hospital Colorado: Allergy Program||Not yet recruiting|
|Aurora, Colorado, United States, 80045|
|Contact: Pascuala Pinedo-Estrada 720-777-8077 firstname.lastname@example.org|
|United States, District of Columbia|
|Children's National Medical Center: Children's Research Institute||Not yet recruiting|
|Washington, District of Columbia, United States, 20010|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago: Division of Allergy and Immunology||Not yet recruiting|
|Chicago, Illinois, United States, 60611|
|United States, Massachusetts|
|Boston Children's Hospital: Department of Immunology||Not yet recruiting|
|Boston, Massachusetts, United States, 02215|
|United States, New York|
|Icahn School of Medicine at Mount Sinai: Division of Clinical Immunology, Immunology Institute||Recruiting|
|New York, New York, United States, 10029|
|Contact: Paula Busse 212-241-0764 email@example.com|
|Principal Investigator: Paula Busse|
|Columbia University Medical Center: Division of Pediatric Pulmonology||Recruiting|
|New York, New York, United States, 10032|
|Contact: Marcela Pierce 347-344-3000 firstname.lastname@example.org|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center: Asthma Center||Recruiting|
|Cincinnati, Ohio, United States, 45229|
|Contact: Anna-Liisa Vockell 513-356-6102 CAUSEScheduling@cchmc.org|
|Study Chair:||Daniel J. Jackson, M.D.||University of Wisconsin School of Medicine and Public Health; Division of Allergy & Immunology|