Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

Pulmonary Sarcoidosis Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05415137
• Other study ID #: ATYR1923-C-004
• Status: Recruiting
• Phase: Phase 3
• Start date: September 15, 2022
• Est. completion date: January 15, 2025

Study information

• Verified date: January 2024
• Source: aTyr Pharma, Inc.
• Contact: aTyr Pharma Clinical Research
• Phone: 877-215-5731
• Email: clinicaltrials[@]atyrpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 264
• Est. completion date: January 15, 2025
• Est. primary completion date: December 15, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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-a) 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

Related Conditions & MeSH terms

• Pulmonary Sarcoidosis
• Sarcoidosis
• Sarcoidosis, Pulmonary

Intervention

Drug:
• Efzofitimod 3 mg/kg
EfzofitimodIV infusion every 4 weeks for a total of 12 doses
• Efzofitimod 5 mg/kg
EfzofitimodIV infusion every 4 weeks for a total of 12 doses
• Placebo
Placebo IV infusion every 4 weeks for a total of 12 doses

Locations

Country	Name	City	State
Brazil	aTyr Investigative Site	Curitiba	PR
Brazil	aTyr Investigative Site	Porto Alegre	RS
Brazil	aTyr Investigative Site	Porto Alegre
Brazil	aTyr Investigative Site	Salvador	BA
Brazil	aTyr Investigative Site	São Bernardo Do Campo
Brazil	aTyr Investigative Site	São Paulo
France	aTyr Investigative Site	Bobigny
France	aTyr Investigative Site	La Tronche
France	aTyr Investigative Site	Montpellier
France	aTyr Investigative Site	Paris
France	aTyr Investigative Site	Toulouse
Germany	aTyr Investigative Site	Essen
Germany	aTyr Investigative Site	Freiburg
Germany	aTyr Investigative Site	Hannover
Germany	aTyr Investigative Site	Heidelberg
Italy	aTyr Investigative Site	Catania
Italy	aTyr Investigative Site	Florence
Italy	aTyr Investigative Site	Forlì
Italy	aTyr Investigative Site	Milan
Italy	aTyr Investigative Site	Napoli
Italy	aTyr Investigative Site	Padua
Italy	aTyr Investigative Site	Rome
Italy	aTyr Investigative Site	Siena
Italy	aTyr Investigative Site	Trieste
Japan	Kyorin Investigative Site	Aomori
Japan	Kyorin Investigative Site	Fukuoka
Japan	Kyorin Investigative Site	Hokkaido
Japan	Kyorin Investigative Site	Hokkaido
Japan	Kyorin Investigative Site	Hyogo
Japan	Kyorin Investigative Site	Kanagawa
Japan	Kyorin Investigative Site	Kanagawa
Japan	aTyr Investigative Site	Kumamoto
Japan	Kyorin Investigative Site	Kumamoto
Japan	Kyorin Investigative Site	Miyagi
Japan	Kyorin Investigative Site	Okayama
Japan	Kyorin Investigative Site	Osaka
Japan	Kyorin Investigative Site	Shimane
Japan	Kyorin Investigative Site	Shizuoka
Japan	Kyorin Investigative Site	Tochigi
Japan	Kyorin Investigative Site	Tokyo
Japan	Kyorin Investigative Site	Tokyo
Netherlands	aTyr Investigative Site	Amsterdam
Netherlands	aTyr Investigative Site	Eindhoven
Netherlands	aTyr Investigative Site	Nieuwegein
Puerto Rico	aTyr Investigative Site	Guaynabo
Spain	aTyr Investigative Site	Barcelona
Spain	aTyr Investigative Site	Barcelona
Spain	aTyr Investigative Site	Madrid
Spain	aTyr Investigative Site	Madrid
Spain	aTyr Investigative Site	Santander
Spain	aTyr Investigative Site	Valencia
United Kingdom	aTyr Investigative Site	Birmingham
United Kingdom	aTyr Investigative Site	Cambridge
United Kingdom	aTyr Investigative Site	Cottingham
United Kingdom	aTyr Investigative Site	Coventry
United Kingdom	aTyr Investigative Site	London
United Kingdom	aTyr Investigative Site	London
United States	aTyr Investigative Site	Ada	Michigan
United States	aTyr Investigative Site	Albany	New York
United States	aTyr Investigative Site	Atlanta	Georgia
United States	aTyr Investigative Site	Atlanta	Georgia
United States	aTyr Investigative Site	Baltimore	Maryland
United States	aTyr Investigative Site	Birmingham	Alabama
United States	aTyr Investigative Site	Boston	Massachusetts
United States	aTyr Investigative Site	Charleston	South Carolina
United States	aTyr Investigative Site	Chicago	Illinois
United States	aTyr Investigative Site	Chicago	Illinois
United States	aTyr Investigative Site	Cincinnati	Ohio
United States	aTyr Investigative Site	Cleveland	Ohio
United States	aTyr Investigative Site	Dallas	Texas
United States	aTyr Investigative Site	Dallas	Texas
United States	aTyr Investigative Site	Denver	Colorado
United States	aTyr Investigative Site	Detroit	Michigan
United States	aTyr Investigative Site	Dickson	Tennessee
United States	aTyr Investigative Site	Doral	Florida
United States	aTyr Investigative Site	Durham	North Carolina
United States	aTyr Investigative Site	Falls Church	Virginia
United States	aTyr Investigative Site	Gainesville	Florida
United States	aTyr Investigative Site	Greenville	North Carolina
United States	aTyr Investigative Site	Houston	Texas
United States	aTyr Investigative Site	Jackson	Mississippi
United States	aTyr Investigative Site	Kansas City	Kansas
United States	aTyr Investigative Site	Los Angeles	California
United States	aTyr Investigative Site	Louisville	Kentucky
United States	aTyr Investigative Site	Miami Lakes	Florida
United States	aTyr Investigative Site	Middletown	New York
United States	aTyr Investigative Site	Minneapolis	Minnesota
United States	aTyr Investigative Site	Nashville	Tennessee
United States	aTyr Investigative Site	New Orleans	Louisiana
United States	aTyr Investigative Site	Newport Beach	California
United States	aTyr Investigative Site	Oklahoma City	Oklahoma
United States	aTyr Investigative Site	Philadelphia	Pennsylvania
United States	aTyr Investigative Site	Phoenix	Arizona
United States	aTyr Investigative Site	Portland	Oregon
United States	aTyr Investigative Site	Richmond	Virginia
United States	aTyr Investigative Site	Rochester	Minnesota
United States	aTyr Investigative Site	Royal Oak	Michigan
United States	aTyr Investigative Site	Sacramento	California
United States	aTyr Investigative Site	Saint Louis	Missouri
United States	aTyr Investigative Site	Salt Lake City	Utah
United States	aTyr Investigative Site	Tampa	Florida
United States	aTyr Investigative Site	Valencia	California
United States	aTyr Investigative Site	Washington	District of Columbia
United States	aTyr Investigative Site	Weston	Florida

Sponsors (2)

Lead Sponsor	Collaborator
aTyr Pharma, Inc.	Kyorin Pharmaceutical Co.,Ltd

Countries where clinical trial is conducted

United States,  Brazil,  France,  Germany,  Italy,  Japan,  Netherlands,  Puerto Rico,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper		Baseline to Week 48
Secondary	Annual rate of change in absolute value of Forced vital capacity (FVC)		Baseline to Week 48
Secondary	Percent change from baseline in mean daily OCS dose post-taper		Baseline to Week 48
Secondary	Change from baseline in King's Sarcoidosis Questionnaire (KSQ)-Lung score		Baseline to Week 48