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Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

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ClinicalTrials.gov Identifier: NCT05415137
Recruitment Status : Recruiting
First Posted : June 10, 2022
Last Update Posted : June 3, 2024
Sponsor:
Collaborator:
Kyorin Pharmaceutical Co.,Ltd
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE June 8, 2022
First Posted Date  ICMJE June 10, 2022
Last Update Posted Date June 3, 2024
Actual Study Start Date  ICMJE September 15, 2022
Estimated Primary Completion Date December 15, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE
 (submitted: June 8, 2022)
Change from baseline in mean daily OCS dose post-taper [ Time Frame: Baseline to Week 48 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Sarcoidosis
Intervention  ICMJE
  • 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  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 8, 2022)
264
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 15, 2025
Estimated Primary Completion Date December 15, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: aTyr Pharma Clinical Research 877-215-5731 clinicaltrials@atyrpharma.com
Listed Location Countries  ICMJE Brazil,   France,   Germany,   Italy,   Japan,   Netherlands,   Puerto Rico,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05415137
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: Undecided
Current Responsible Party aTyr Pharma, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE aTyr Pharma, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Kyorin Pharmaceutical Co.,Ltd
Investigators  ICMJE
Study Director: Lisa Carey aTyr Pharma, Inc.
PRS Account aTyr Pharma, Inc.
Verification Date May 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP