Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

January 31, 2024 updated by: aTyr Pharma, Inc.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

264

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Brazil
      • Porto Alegre, Brazil
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
      • São Bernardo Do Campo, Brazil, 09780-000
      • São Paulo, Brazil, 05403-900
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • BA
      • Salvador, BA, Brazil, 40220-005
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • PR
      • Curitiba, PR, Brazil, 80060-150
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • RS
      • Porto Alegre, RS, Brazil, 90020-090
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
  • France
      • Bobigny, France, 93000
      • La Tronche, France, 38700
      • Montpellier, France, 34295
      • Paris, France, 75015
      • Toulouse, France, 31000
  • Germany
      • Essen, Germany, 45122
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
      • Freiburg, Germany, 79106
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
      • Hannover, Germany, 30625
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
      • Heidelberg, Germany, 69126
  • Italy
      • Catania, Italy, 95124
      • Florence, Italy, 50134
      • Forlì, Italy, 47121
      • Milan, Italy, 20123
      • Napoli, Italy, 80131
      • Padua, Italy, 35128
      • Rome, Italy, 00168
      • Siena, Italy, 53100
      • Trieste, Italy, 34149
  • Japan
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
      • Eindhoven, Netherlands, 5623 EJ
      • Nieuwegein, Netherlands, 3435CM
  • Puerto Rico
      • Guaynabo, Puerto Rico, 00968
        • Withdrawn
        • aTyr Investigative Site
  • Spain
      • Barcelona, Spain, 08036
      • Barcelona, Spain, 08907
      • Madrid, Spain, 28006
      • Madrid, Spain, 28031
      • Santander, Spain, 39008
      • Valencia, Spain, 46010
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
      • Cambridge, United Kingdom, CB233RE
      • Cottingham, United Kingdom, HU16 5JQ
      • Coventry, United Kingdom, CV2 2DX
      • London, United Kingdom, SE59RS
      • London, United Kingdom, sW3 6NP
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
    • Arizona
      • Phoenix, Arizona, United States, 85027
    • California
      • Los Angeles, California, United States, 90033
      • Newport Beach, California, United States, 92663
        • Terminated
        • aTyr Investigative Site
      • Sacramento, California, United States, 95817
      • Valencia, California, United States, 23823
    • Colorado
      • Denver, Colorado, United States, 80206
    • District of Columbia
      • Washington, District of Columbia, United States, 20059
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • Florida
      • Doral, Florida, United States, 33166
        • Withdrawn
        • aTyr Investigative Site
      • Gainesville, Florida, United States, 32610
      • Miami Lakes, Florida, United States, 33014
        • Withdrawn
        • aTyr Investigative Site
      • Tampa, Florida, United States, 33606
      • Weston, Florida, United States, 33331
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30322
      • Atlanta, Georgia, United States, 30342
    • Illinois
      • Chicago, Illinois, United States, 60611
      • Chicago, Illinois, United States, 60612
    • Kansas
      • Kansas City, Kansas, United States, 48202
    • Kentucky
      • Louisville, Kentucky, United States, 40202
    • Louisiana
      • New Orleans, Louisiana, United States, 70115
    • Maryland
      • Baltimore, Maryland, United States, 21224
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
    • Michigan
      • Ada, Michigan, United States, 49301
      • Detroit, Michigan, United States, 48202
      • Royal Oak, Michigan, United States, 48073
    • Minnesota
      • Minneapolis, Minnesota, United States, 55445
      • Rochester, Minnesota, United States, 55905
    • Mississippi
      • Jackson, Mississippi, United States, 38216
    • Missouri
      • Saint Louis, Missouri, United States, 63110
    • New York
      • Albany, New York, United States, 12208
      • Middletown, New York, United States, 10941
        • Not yet recruiting
        • aTyr Investigative Site
        • Contact:
    • North Carolina
      • Durham, North Carolina, United States, 27710
      • Greenville, North Carolina, United States, 27834
    • Ohio
      • Cincinnati, Ohio, United States, 45267
      • Cleveland, Ohio, United States, 44195
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
    • Oregon
      • Portland, Oregon, United States, 97220
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19140
    • South Carolina
      • Charleston, South Carolina, United States, 29425-0630
    • Tennessee
      • Dickson, Tennessee, United States, 37055
      • Nashville, Tennessee, United States, 37204
    • Texas
      • Dallas, Texas, United States, 75246
      • Dallas, Texas, United States, 75390
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84108
    • Virginia
      • Falls Church, Virginia, United States, 22042
      • Richmond, Virginia, United States, 23298

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Placebo IV infusion every 4 weeks for a total of 12 doses
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper
Time Frame: Baseline to Week 48
Baseline to Week 48

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

aTyr Pharma, Inc.

Collaborators

Kyorin Pharmaceutical Co.,Ltd

Investigators

  • Study Director: Lisa Carey, aTyr Pharma, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 15, 2022

Primary Completion (Estimated)

December 15, 2024

Study Completion (Estimated)

January 15, 2025

Study Registration Dates

First Submitted

June 8, 2022

First Submitted That Met QC Criteria

June 8, 2022

First Posted (Actual)

June 10, 2022

Study Record Updates

Last Update Posted (Estimated)

February 2, 2024

Last Update Submitted That Met QC Criteria

January 31, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATYR1923-C-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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