Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

Sponsor

aTyr Pharma, Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05415137

Collaborator

Kyorin Pharmaceutical Co.,Ltd (Industry)

264

Enrollment

Location

Arms

Anticipated Duration (Months)

9.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Pulmonary Sarcoidosis	Drug: Efzofitimod 3 mg/kg Drug: Efzofitimod 5 mg/kg Drug: Placebo	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

264 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Sep 15, 2022

Anticipated Primary Completion Date :

Dec 15, 2024

Anticipated Study Completion Date :

Jan 15, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Efzofitimod 3 mg/kg	Drug: Efzofitimod 3 mg/kg EfzofitimodIV infusion every 4 weeks for a total of 12 doses Other Names: ATYR1923 KRP-R120
Experimental: Efzofitimod 5 mg/kg	Drug: Efzofitimod 5 mg/kg EfzofitimodIV infusion every 4 weeks for a total of 12 doses Other Names: ATYR1923 KRP-R120
Placebo Comparator: Placebo	Drug: Placebo Placebo IV infusion every 4 weeks for a total of 12 doses

Arm

Intervention/Treatment

Experimental: Efzofitimod 3 mg/kg

Drug: Efzofitimod 3 mg/kg

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Other Names:

ATYR1923

KRP-R120

Experimental: Efzofitimod 5 mg/kg

Drug: Efzofitimod 5 mg/kg

EfzofitimodIV infusion every 4 weeks for a total of 12 doses

Other Names:

ATYR1923

KRP-R120

Placebo Comparator: Placebo

Drug: Placebo

Placebo IV infusion every 4 weeks for a total of 12 doses

Outcome Measures

Primary Outcome Measures

Change from baseline in mean daily OCS dose post-taper [Baseline to Week 48]

Secondary Outcome Measures

Annual rate of change in absolute value of Forced vital capacity (FVC) [Baseline to Week 48]
Percent change from baseline in mean daily OCS dose post-taper [Baseline to Week 48]
Change from baseline in King's Sarcoidosis Questionnaire (KSQ)-Lung score [Baseline to Week 48]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Confirmed diagnosis of pulmonary sarcoidosis for at least 6 months, defined by the following criteria: 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 with a starting dose between ≥ 7.5 and ≤ 25 mg/day.
Body weight ≥ 40 kg and < 160 kg

Exclusion Criteria:

Treatment with > 1 oral 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% at Screening; FVC % predicted < 50% and KSQ-Lung score < 30
Clinically significant pulmonary hypertension requiring treatment with vasodilators
Patients with cardiac sarcoidosis, neurosarcoidosis, or renal sarcoidosis
Clinically significant cutaneous and ocular sarcoidosis
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 baseline