Study Assessing Long-teRm Outcomes of dupiluMAb (DUPIXENT®) Treatment in Adult Patients With Chronic Rhinosinusitis With Nasal Polyposis (CRSwNP) (AROMA)

• ClinicalTrials.gov Identifier: NCT04959448
• Recruitment Status: Recruiting
• First Posted: July 13, 2021
• Last Update Posted: June 5, 2023
• Sponsor: Regeneron Pharmaceuticals
• Collaborator: Sanofi
• Information provided by (Responsible Party): Regeneron Pharmaceuticals

Tracking Information

• First Submitted Date: June 21, 2021
• First Posted Date: July 13, 2021
• Last Update Posted Date: June 5, 2023
• Actual Study Start Date: August 12, 2021
• Estimated Primary Completion Date: August 17, 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 8, 2021)

• Baseline Patient Characteristics [ Time Frame: Baseline up to 3 years ]
  Demographics includes but is not limited to gender, age, ethnicity, height, weight, BMI, education, current employment status, physician information.
• Baseline Disease Characteristics [ Time Frame: Baseline up to 3 years ]
  Medical History includes but is not limited to history of CRSwNP, comorbid conditions, age at CRSwNP diagnosis, and baseline measurements of effectiveness variables.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 8, 2021)

• DUPIXENT® Treatment Characteristics [ Time Frame: Baseline up to 3 years ]
  Including but not limited to: usage, dosage, adherence, interruption, place (home or clinic) and frequency of administration
• CRSwNP Treatment Characteristics [ Time Frame: Baseline up to 3 years ]
  Including but not limited to: usage, dosage, adherence, duration and frequency
• Incidence and severity of adverse events (AEs) [ Time Frame: Baseline up to 3 years ]
• Participant Assessment: Total symptom score (TSS), including sub-scores (nasal congestion [NC] and loss of smell [LOS]) [ Time Frame: Baseline up to 3 years ]
  The TSS is a composite score (ranging between 0 and 9) and consisting of the sum of the following symptoms assessed daily: nasal congestion/obstruction, decreased/loss of sense of smell, rhinorrhea (average of anterior/posterior nasal discharge). Higher scores indicate greater symptom burden.
• Physician Assessment: Change in University of Pennsylvania Smell Identification Test (UPSIT) score [ Time Frame: Baseline up to 3 years ]
  The UPSIT (UPSIT 40-odorant test) is a rapid and easy-to-administer method to quantitatively assess human olfactory function.
• Physician Assessment: CT-Lund-Mackay Score [ Time Frame: Baseline up to 3 years ]
  The LMK system is scoring based on CT imaging of the sinuses with points given for degree of opacification: 0 = no opacification, 1 = partial opacification, 2 = total opacification. These points are then applied to the maxillary, anterior ethmoid, posterior ethmoid, sphenoid, frontal sinus on each side. The ostiomeatal complex is graded as 0 = not occluded, or 2 = occluded. The sum of these derives a maximum score of 12 per side and 24 in total.
• Physician Assessment: Nasal Peak Inspiratory Flow (PNIF) [ Time Frame: Baseline up to 3 years ]
  Nasal peak inspiratory flow (NPIF) is a measure of the air flow through both nasal cavities during forced inspiration expressed in liters per minute.
• Physician Assessment: - Forced Expiratory Volume in 1 second (FEV1) [ Time Frame: Baseline up to 3 years ]
  Forced Expiratory Volume in 1 second (FEV1) is the amount of air which can be forcibly exhaled in the first second of a forced exhalation
• Physician Assessment: Fractional exhaled Nitric Oxide (FeNO) [ Time Frame: Baseline up to 3 Years ]
  FeNO (Fractional exhaled Nitric Oxiide) is analyzed using a respiratory flow rate of 50 mL/second and reported in ppb.
• Participant Assessment: Asthma Control Questionnaire, 6-item (ACQ-6) [ Time Frame: Baseline up to 3 Years ]
  The ACQ-6 assesses asthma control with scores ranging from 0 (fully controlled; 6=severely uncontrolled).
• Participant Assessment: Mini Asthma Quality of Life Questionnaire (MiniAQLQ) [ Time Frame: Baseline up to 3 Years ]
  The MiniAQLQ is assesses the impact of asthma on health-related quality of life with scores ranging from 1 (maximum impairment) to 7 (no impairment).
• Participant Assessment: Allergic Rhinitis (AR) using AR Visual Analog Scale (VAS) [ Time Frame: Baseline up to 3 Years ]
  The AR-VAS assesses severity of rhinosinusitis symptoms on a 0 (not troublesome) to 10 (worst thinkable troublesome) scale.
• Participant Assessment: Mini Rhinoconjunctivitis Quality of Life Questionnaire (MiniRQLQ) [ Time Frame: Baseline up to 3 Years ]
  The MiniRQLQ is an abbreviated version of the RQLQ(S)+12 which assesses health-related quality of life associated with perennial or seasonal allergic rhinitis with scores ranging from 0 (not troubled) to 6 (extremely troubled).
• Participant Assessment: Sino-Nasal Outcome Test (SNOT-22) [ Time Frame: Baseline up to 3 Years ]
  The SNOT-22 assesses the impact of chronic rhinosinusitis on health-related quality of life with scores ranging from 0 to 110; lower scores representing better health related quality of life.
• Participant Assessment: Patient Oriented Eczema Measure (POEM) [ Time Frame: Baseline up to 3 Years ]
  The POEM assesses symptoms of dryness, itching, flaking, cracking, sleep loss, bleeding, and weeping with scores ranging from 0 to 28; higher scores indicate greater symptom burden.
• Participant Assessment: Work Productivity and Activity Impairment Questionnaire for asthma (WPAI-CRSwNP) [ Time Frame: Baseline up to 3 Years ]
  The WPAI-CRSwNP measures impairments in work and activities associated with CRSwNP. Scores are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity.
• Physician Assessment: Changes of Healthcare Utilization [ Time Frame: Baseline up to 3 years ]
  Resource use collected from medical, hospital, and pharmacy records
• Participant Assessment: Short Form 12 [ Time Frame: Baseline up to 3 years ]
  The SF-12 assesses physical functioning, social functioning, role limitations due to physical problems, role limitations due to emotional problems, mental health, energy/vitality, pain, and general health perception with scores ranging from 0 to 100; lower scores represent greater quality of life impairment.
• Participant Assessment: European Quality of Life 5-Dimensions, 5-Level Questionnaire (EQ-5D-5L) [ Time Frame: Baseline up to 3 years ]
  The EQ 5D-5L descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression and the EQ-VAS records the respondent's self rated health on a scale of 0 (worst imaginable health state) to 100 (best imaginable health state).
• Participant Assessment: Global Impression for Symptom Severity, Treatment Satisfaction (Global Patient Assessment) [ Time Frame: Baseline up to 3 years ]
  The Global Patient Assessment is a 2-component questionnaire on symptom severity over the past week and the patient's overall satisfaction with their CRSwNP treatment.
• Physician Assessment: Global Impression for Disease Severity (Global Physician Assessment) [ Time Frame: Baseline up to 3 years ]
  The Global Physician Assessment is a 1-item question asking physicians to rate the severity of their patient's CRSwNP.

• 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: Study Assessing Long-teRm Outcomes of dupiluMAb (DUPIXENT®) Treatment in Adult Patients With Chronic Rhinosinusitis With Nasal Polyposis (CRSwNP)
• Official Title: Assessing Long-Term Outcomes of DUPIXENT® Treatment in Patients With Chronic Rhinosinusitis With Nasal Polyposis (AROMA)

Brief Summary

The primary objectives of the study are:

• To longitudinally characterize the long-term effectiveness of DUPIXENT® through assessment of patient-reported symptoms, Health-Related Quality of Life (HRQoL) related to Chronic rhinosinusitis with nasal polyposis (CRSwNP) and other type 2 comorbidities, and their change over-time.
• To characterize patients who receive DUPIXENT® for CRSwNP in a real-world setting with respect to their medical history, demographic and disease characteristics, and type 2 comorbidities

The secondary objectives of the study are:

• To characterize real-world utilization of DUPIXENT® for patients with CRSwNP
• To collect patient and physician global assessment of disease severity and treatment satisfaction for patients receiving DUPIXENT® for CRSwNP
• To collect long-term safety data for patients receiving DUPIXENT® for CRSwNP

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Patients who are initiating treatment with DUPIXENT® for CRSwNP according to the country-specific prescribing information.
• Condition: Chronic Rhinosinusitis With Nasal Polyposis

Intervention

Drug: DUPIXENT®

No investigational agents will be provided. All treatments will be prescribed at the discretion of the study physician and other healthcare providers.

Other Names:

• dupilumab
• REGN668
• SAR231893

• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: July 8, 2021): 1000
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: August 17, 2026
• Estimated Primary Completion Date: August 17, 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Key Inclusion Criteria:

• All patients who are newly initiated on DUPIXENT® for the treatment of CRSwNP according to the respective prescribing information (Product Label or SmPC)
• Willing and able to comply with clinic visits and study-related procedures as per protocol
• Provide informed consent signed by study patient or legally acceptable representative
• Able to understand and complete study-related questionnaires as per protocol

Key Exclusion Criteria:

• Patients who have a contraindication to DUPIXENT® according to the country-specific prescribing information
• Any previous treatment with DUPIXENT® for any condition
• Any condition that, in the opinion of the investigator, may interfere with the patient's ability to participate in the study per protocol
• Participation in an ongoing interventional or observational study that might, in the treating physician's opinion, influence the assessments for the current study per protocol

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Clinical Trials Administrator; 844-734-6643; clinicaltrials@regeneron.com

• Listed Location Countries: Canada, Germany, Italy, Japan, United States

Administrative Information

• NCT Number: NCT04959448
• Other Study ID Numbers: R668-cSNP-2072; EUPAS41656 ( Other Identifier: ENCePP )
• 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: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
• Supporting Materials: Study Protocol
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Informed Consent Form (ICF)
• Supporting Materials: Clinical Study Report (CSR)
• Supporting Materials: Analytic Code
• Time Frame: When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
• URL: https://vivli.org/

• Current Responsible Party: Regeneron Pharmaceuticals
• Original Responsible Party: Same as current
• Current Study Sponsor: Regeneron Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Sanofi

Investigators

• Study Director: Clinical Trial Management; Regeneron Pharmaceuticals

• PRS Account: Regeneron Pharmaceuticals
• Verification Date: December 2022