Clinical trial NCT03968978: Tezepelumab Home Use Study
Official title: A Multicenter, Randomized, Open-label, Parallel Group, Functionality, and Performance Study of an Accessorized Pre-filled Syringe and Autoinjector With Home-administered Subcutaneous Tezepelumab in Adolescent and Adult Subjects With Severe Asthma (PATH-HOME)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Asthma
Keywords: Asthma, Severe Uncontrolled Asthma
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
Collaborators: name: Amgen
First posted: 2019-05-30
Enrollment: 216
Sex: ALL
Minimum age (years): 12
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Subjects will be randomized 1:1 to either an accessorized pre-filled syringe or an autoinjector. Both will be administered 210 mg tezepelumab subcutaneously.
Primary purpose: TREATMENT

Brief summary: This is a multicenter, randomized, open-label, parallel-group study designed to assess healthcare provider and subject/caregiver reported functionality and performance of a single-use accessorized pre-filled syringe (APFS) or autoinjector (AI) with a fixed 210 mg dose of tezepelumab administered subcutaneously in the clinic and in an at-home setting.

Detailed description: The study will consist of a screening/run-in period of up to 2 weeks and a treatment period of 24 weeks, followed by a post-treatment follow-up period of 12 weeks. During the treatment period, one dose of 210 mg tezepelumab will be administered via a single-use APFS or AI subcutaneously (SC) every 4 weeks (Q4W) starting at Visit 2 (Week 0) until Visit 7 (Week 20). Subjects will be administered tezepelumab at the site during Visits 2 (Week 0), 3 (Week 4), 4 (Week 8) and 7 (Week 20). At-home administration of tezepelumab will occur during Visit 5 (Week 12) and Visit 6 (Week 16). Each device will be assessed separately using descriptive presentations.

Eligibility criteria: Inclusion Criteria:

* Male or female, age 12 to 80 years.
* Documented physician-diagnosed asthma for at least 12 months.
* Evidence of asthma as documented by post BD (albuterol/salbutamol) reversibility of FEV1 ≥ 12% AND ≥200 mL (15-60 min after administration of 4 puffs of albuterol/salbutamol), documented either: in the previous 12 months prior to V1, OR demonstrated at V1, V1A, or at V2.
* Documented history of current treatment with medium- or high-dose ICS for at least 6 months and at least one additional asthma controller medication according to standard practice of care. ICS dose must be greater than or equal to 500 μg/day fluticasone propionate dry powder formulation or equivalent daily.
* Morning pre-BD FEV1 of \>50% predicted normal at Visit 1, Visit 1A, or Visit 2.

Exclusion Criteria:

* Clinically important pulmonary or systemic diseases other than asthma.
* History of cancer except basal cell carcinoma, squamous cell carcinoma, or in situ carcinoma of the cervix within 12 months prior to Visit 1.
* Acute upper or lower respiratory infection requiring antibiotics or antiviral medications finalized \<2 weeks before Visit 1 or during screening/run-in period.
* A helminth parasitic infection diagnosed within 6 months that is untreated or is unresponsive to the standard of care.
* Smoking history of ≥10 pack years, (includes vaping and e-cigarettes)
* History of chronic alcohol or drug abuse.
* Tuberculosis requiring treatment within 12 months prior to V1.
* History of HIV, Hepatitis B or Hepatitis C.
* Receipt of any marketed or investigational biologic agent within 4 months or 5 half-lives (whichever is longer) prior to visit 1 or receipt of any investigational non-biologic agent within 30 days or 5 half-lives.
* Bronchial thermoplasty in 24 months prior to V1.
* Anaphylaxis or documented immune complex disease (Type III hypersensitivity reactions) to any biologic therapy.
* Evidence of active liver disease (e.g. jaundice, AST, ALT or ALP \>2 times upper limit of normal), ongoing liver disease or inexplicably elevated liver chemistry values.
* Pregnant, breastfeeding or lactating women.
* Non-leukocyte depleted whole blood transfusion in 120 days prior to visit 1.

Primary outcomes:
measure: Proportions of HCPs and Subjects/Caregivers Who Successfully Administered Tezepelumab in Clinic or at Home by Device Type
timeFrame: Week 0, Week 4, Week 8, Week 12, Week 16, Week 20
Secondary outcomes:
measure: Proportions of Used/Returned Devices That Pass Functional Tests and Visual Inspection and Showed no Evidence of Malfunction
timeFrame: Week 0, Week 4, Week 8, Week 12, Week 16, Week 20
measure: Proportions of Devices That Have Been Reported as Malfunctioning (Product Complaints)
timeFrame: Week 0, Week 4, Week 8, Week 12, Week 16, Week 20
measure: Change From Baseline in Asthma Control Questionnaire-6 (ACQ-6) Score
timeFrame: Baseline (Week 0), Week 4, Week 8, Week 12, Week 16, Week 20 and Week 24
measure: Serum Trough Concentrations
timeFrame: Baseline (Week 0), Week 4, Week 20 and Week 24 (EOT)
measure: Anti-drug Antibodies (ADA)
timeFrame: Pre-treatment on dosing days until end of follow-up (Week 36) per protocol
Other outcomes:
Locations:
city: Hoover
country: United States
facility: Research Site
state: Alabama
city: Gilbert
country: United States
facility: Research Site
state: Arizona
city: Northridge
country: United States
facility: Research Site
state: California
city: Palm Desert
country: United States
facility: Research Site
state: California
city: Westminster
country: United States
facility: Research Site
state: California
city: Tampa
country: United States
facility: Research Site
state: Florida
city: Savannah
country: United States
facility: Research Site
state: Georgia
city: Omaha
country: United States
facility: Research Site
state: Nebraska
city: Northfield
country: United States
facility: Research Site
state: New Jersey
city: Cincinnati
country: United States
facility: Research Site
state: Ohio
city: Edmond
country: United States
facility: Research Site
state: Oklahoma
city: Medford
country: United States
facility: Research Site
state: Oregon
city: Dallas
country: United States
facility: Research Site
state: Texas
city: McKinney
country: United States
facility: Research Site
state: Texas
city: San Antonio
country: United States
facility: Research Site
state: Texas
city: San Antonio
country: United States
facility: Research Site
state: Texas
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Ajax
country: Canada
facility: Research Site
state: Ontario
city: Burlington
country: Canada
facility: Research Site
state: Ontario
city: Mississauga
country: Canada
facility: Research Site
state: Ontario
city: Ottawa
country: Canada
facility: Research Site
state: Ontario
city: Windsor
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Trois-Rivieres
country: Canada
facility: Research Site
state: Quebec
city: Quebec
country: Canada
facility: Research Site
city: Quebec
country: Canada
facility: Research Site
city: Chuo-ku
country: Japan
facility: Research Site
city: Fukuoka-shi
country: Japan
facility: Research Site
city: Białystok
country: Poland
facility: Research Site
city: Kraków
country: Poland
facility: Research Site
city: Poznań
country: Poland
facility: Research Site
city: Poznań
country: Poland
facility: Research Site
city: Strzelce Opolskie
country: Poland
facility: Research Site
city: Tarnów
country: Poland
facility: Research Site
city: Wieluń
country: Poland
facility: Research Site
city: Wrocław
country: Poland
facility: Research Site
Study officials:
affiliation: Clinical Research Trials of Florida, Inc.
name: Sady A Alpizar, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Alpizar S, Megally A, Chen C, Raj A, Downie J, Colice G. Functionality and Performance of an Accessorized Pre-Filled Syringe and an Autoinjector for At-Home Administration of Tezepelumab in Patients with Severe, Uncontrolled Asthma. J Asthma Allergy. 2021 Apr 19;14:381-392. doi: 10.2147/JAA.S305114. eCollection 2021.