Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

• ClinicalTrials.gov Identifier: NCT05415137
• Recruitment Status: Recruiting
• First Posted: June 10, 2022
• Last Update Posted: February 2, 2024
• Sponsor: aTyr Pharma, Inc.
• Collaborator: Kyorin Pharmaceutical Co.,Ltd
• Information provided by (Responsible Party): aTyr Pharma, Inc.

Tracking Information

• First Submitted Date: June 8, 2022
• First Posted Date: June 10, 2022
• Last Update Posted Date: February 2, 2024
• Actual Study Start Date: September 15, 2022
• Estimated Primary Completion Date: December 15, 2024 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: September 7, 2022): Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper [ Time Frame: Baseline to Week 48 ]
• Original Primary Outcome Measures (submitted: June 8, 2022): Change from baseline in mean daily OCS dose post-taper [ Time Frame: Baseline to Week 48 ]

Current Secondary Outcome Measures (submitted: June 8, 2022)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Pulmonary Sarcoidosis

Intervention

• Drug: Efzofitimod 3 mg/kg
  EfzofitimodIV infusion every 4 weeks for a total of 12 doses
  Other Names:

  • ATYR1923
  • KRP-R120
• Drug: Efzofitimod 5 mg/kg
  EfzofitimodIV infusion every 4 weeks for a total of 12 doses
  Other Names:

  • ATYR1923
  • KRP-R120
• Drug: Placebo
  Placebo IV infusion every 4 weeks for a total of 12 doses

Study Arms

• Experimental: Efzofitimod 3 mg/kg
  Intervention: Drug: Efzofitimod 3 mg/kg
• Experimental: Efzofitimod 5 mg/kg
  Intervention: Drug: Efzofitimod 5 mg/kg
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: June 8, 2022): 264
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: January 15, 2025
• Estimated Primary Completion Date: December 15, 2024 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: aTyr Pharma Clinical Research; 877-215-5731; clinicaltrials@atyrpharma.com

• Listed Location Countries: Brazil, France, Germany, Italy, Japan, Netherlands, Puerto Rico, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT05415137
• Other Study ID Numbers: ATYR1923-C-004
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: aTyr Pharma, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: aTyr Pharma, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Kyorin Pharmaceutical Co.,Ltd

Investigators

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

• PRS Account: aTyr Pharma, Inc.
• Verification Date: January 2024