Clinical trial NCT05415137: Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Pulmonary Sarcoidosis
Keywords: Pulmonary Sarcoidosis
Sarcoidosis
Granuloma
Inflammation
Lymphoproliferative Disorders
Interstitial Lung Disease
Neuropilin-2
Steroids
Oral corticosteroids
Immunomodulatory
tRNA Synthetase
ATYR1923
KRP-R120
Efzofitimod
Fibrosis
Lead sponsor: aTyr Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Kyorin Pharmaceutical Co.,Ltd
First posted: 2022-06-10
Enrollment: 268
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a multicenter, randomized, double-blind, placebo-controlled, study comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo after 48 weeks of treatment. This study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid (OCS), with or without immunosuppressant therapy.


Eligibility criteria: Inclusion Criteria:

* Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence
* Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70
* Patients must be receiving treatment with OCS of ≥ 3 months at Day 1 with a starting dose between ≥ 7.5 and ≤ 25 mg/day ≥ 4 weeks prior to Day 1.
* Body weight ≥ 40 kg and \< 160 kg

Exclusion Criteria:

* Treatment with \> 1 immunosuppressant therapy
* Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
* Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis \> 20% within the last 12 months; FVC percent predicted (FVCPP) \< 50% and KSQ-Lung score \< 30
* In the opinion of the investigator, clinically significant pulmonary hypertension
* Patients with active cardiac, neuro, or renal sarcoidosis requiring organ-specific therapy in the past 2 years
* Patients with cutaneous or ocular sarcoidosis, which in the opinion of the Investigator, are at risk for exacerbation, necessitating OCS rescue or other systemic therapy
* History of Addisonian symptoms that precluded previous OCS taper attempts
* Is an active, heavy smoker of tobacco/nicotine-containing products
* History of anti-synthetase syndrome or Jo-1 positive at Screening
* Patients with active tuberculosis or those currently undergoing treatment for tuberculosis

Primary outcomes:
measure: Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
timeFrame: Baseline to Week 48
Secondary outcomes:
measure: Change from baseline in KSQ-Lung score at Week 48
timeFrame: Baseline to Week 48
measure: Steroid withdrawal rate
timeFrame: Baseline to Week 48
measure: Change from baseline in absolute value of FVC at Week 48
timeFrame: Baseline to Week 48
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: aTyr Investigative Site
state: Alabama
city: Phoenix
country: United States
facility: aTyr Investigative Site
state: Arizona
city: Los Angeles
country: United States
facility: aTyr Investigative Site
state: California
city: Sacramento
country: United States
facility: aTyr Investigative Site
state: California
city: Denver
country: United States
facility: aTyr Investigative Site
state: Colorado
city: Washington
country: United States
facility: aTyr Investigative Site
state: District of Columbia
city: Gainesville
country: United States
facility: aTyr Investigative Site
state: Florida
city: Tampa
country: United States
facility: aTyr Investigative Site
state: Florida
city: Weston
country: United States
facility: aTyr Investigative Site
state: Florida
city: Atlanta
country: United States
facility: aTyr Investigative Site
state: Georgia
city: Chicago
country: United States
facility: aTyr Investigative Site
state: Illinois
city: Chicago
country: United States
facility: aTyr Investigative Site
state: Illinois
city: Louisville
country: United States
facility: aTyr Investigative Site
state: Kentucky
city: New Orleans
country: United States
facility: aTyr Investigative Site
state: Louisiana
city: Baltimore
country: United States
facility: aTyr Investigative Site
state: Maryland
city: Boston
country: United States
facility: aTyr Investigative Site
state: Massachusetts
city: Ada
country: United States
facility: aTyr Investigative Site
state: Michigan
city: Detroit
country: United States
facility: aTyr Investigative Site
state: Michigan
city: Minneapolis
country: United States
facility: aTyr Investigative Site
state: Minnesota
city: Rochester
country: United States
facility: aTyr Investigative Site
state: Minnesota
city: Jackson
country: United States
facility: aTyr Investigative Site
state: Mississippi
city: Saint Louis
country: United States
facility: aTyr Investigative Site
state: Missouri
city: Albany
country: United States
facility: aTyr Investigative Site
state: New York
city: Middletown
country: United States
facility: aTyr Investigative Site
state: New York
city: Durham
country: United States
facility: aTyr Investigative Site
state: North Carolina
city: Greenville
country: United States
facility: aTyr Investigative Site
state: North Carolina
city: Cincinnati
country: United States
facility: aTyr Investigative Site
state: Ohio
city: Cleveland
country: United States
facility: aTyr Investigative Site
state: Ohio
city: Oklahoma City
country: United States
facility: aTyr Investigative Site
state: Oklahoma
city: Portland
country: United States
facility: aTyr Investigative Site
state: Oregon
city: Philadelphia
country: United States
facility: aTyr Investigative Site
state: Pennsylvania
city: Charleston
country: United States
facility: aTyr Investigative Site
state: South Carolina
city: Dickson
country: United States
facility: aTyr Investigative Site
state: Tennessee
city: Dallas
country: United States
facility: aTyr Investigative Site
state: Texas
city: Dallas
country: United States
facility: aTyr Investigative Site
state: Texas
city: Houston
country: United States
facility: aTyr Investigative Site
state: Texas
city: Salt Lake City
country: United States
facility: aTyr Investigative Site
state: Utah
city: Falls Church
country: United States
facility: aTyr Investigative Site
state: Virginia
city: Richmond
country: United States
facility: aTyr Investigative Site
state: Virginia
city: Salvador
country: Brazil
facility: aTyr Investigative Site
state: BA
city: Porto Alegre
country: Brazil
facility: aTyr Investigative Site
state: RS
city: São Bernardo Do Campo
country: Brazil
facility: aTyr Investigative Site
city: São Paulo
country: Brazil
facility: aTyr Investigative Site
city: São Paulo
country: Brazil
facility: aTyr Investigative Site
city: Bobigny
country: France
facility: aTyr Investigative Site
city: La Tronche
country: France
facility: aTyr Investigative Site
city: Montpellier
country: France
facility: aTyr Investigative Site
city: Paris
country: France
facility: aTyr Investigative Site
city: Toulouse
country: France
facility: aTyr Investigative Site
city: Essen
country: Germany
facility: aTyr Investigative Site
city: Freiburg
country: Germany
facility: aTyr Investigative Site
city: Hannover
country: Germany
facility: aTyr Investigative Site
city: Heidelberg
country: Germany
facility: aTyr Investigative Site
city: Catania
country: Italy
facility: aTyr Investigative Site
city: Florence
country: Italy
facility: aTyr Investigative Site
city: Forlì
country: Italy
facility: aTyr Investigative Site
city: Milan
country: Italy
facility: aTyr Investigative Site
city: Napoli
country: Italy
facility: aTyr Investigative Site
city: Padua
country: Italy
facility: aTyr Investigative Site
city: Rome
country: Italy
facility: aTyr Investigative Site
city: Siena
country: Italy
facility: aTyr Investigative Site
city: Trieste
country: Italy
facility: aTyr Investigative Site
city: Aomori
country: Japan
facility: Kyorin Investigative Site
city: Fukuoka
country: Japan
facility: Kyorin Investigative Site
city: Fukushima
country: Japan
facility: Kyorin Investigative Site
city: Hokkaido
country: Japan
facility: Kyorin Investigative Site
city: Hokkaido
country: Japan
facility: Kyorin Investigative Site
city: Hyōgo
country: Japan
facility: Kyorin Investigative Site
city: Kanagawa
country: Japan
facility: Kyorin Investigative Site
city: Kanagawa
country: Japan
facility: Kyorin Investigative Site
city: Kumamoto
country: Japan
facility: aTyr Investigative Site
city: Miyagi
country: Japan
facility: Kyorin Investigative Site
city: Okayama
country: Japan
facility: Kyorin Investigative Site
city: Osaka
country: Japan
facility: Kyorin Investigative Site
city: Shimane
country: Japan
facility: Kyorin Investigative Site
city: Shizuoka
country: Japan
facility: Kyorin Investigative Site
city: Tokyo
country: Japan
facility: Kyorin Investigative Site
city: Tokyo
country: Japan
facility: Kyorin Investigative Site
city: Nieuwegein
country: Netherlands
facility: aTyr Investigative Site
city: Barcelona
country: Spain
facility: aTyr Investigative Site
city: Barcelona
country: Spain
facility: aTyr Investigative Site
city: Madrid
country: Spain
facility: aTyr Investigative Site
city: Madrid
country: Spain
facility: aTyr Investigative Site
city: Santander
country: Spain
facility: aTyr Investigative Site
city: Valencia
country: Spain
facility: aTyr Investigative Site
city: Birmingham
country: United Kingdom
facility: aTyr Investigative Site
city: Cambridge
country: United Kingdom
facility: aTyr Investigative Site
city: Cottingham
country: United Kingdom
facility: aTyr Investigative Site
city: Coventry
country: United Kingdom
facility: aTyr Investigative Site
city: London
country: United Kingdom
facility: aTyr Investigative Site
city: London
country: United Kingdom
facility: aTyr Investigative Site
Study officials:
affiliation: aTyr Pharma, Inc.
name: Lisa Carey
role: STUDY_DIRECTOR
References: