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Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis

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ClinicalTrials.gov Identifier: NCT03824392
Recruitment Status : Completed
First Posted : January 31, 2019
Last Update Posted : July 22, 2021
Sponsor:
Collaborator:
Foundation for Sarcoidosis Research
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE January 28, 2019
First Posted Date  ICMJE January 31, 2019
Last Update Posted Date July 22, 2021
Actual Study Start Date  ICMJE January 29, 2019
Actual Primary Completion Date June 29, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 29, 2019)
Incidence of treatment emergent adverse events and serious adverse events [ Time Frame: Baseline to Week 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 29, 2019)
  • Total cumulative steroid dose administered over study period [ Time Frame: Baseline to Week 24 ]
  • Number of participants who achieve and maintain the targeted tapered dose of prednisone 5 mg/kg day [ Time Frame: Baseline to Week 24 ]
  • Exposure response analysis comparing steroid dose area under the curve (AUC) with ATYR1923 PK parameters [ Time Frame: Baseline to Week 24 ]
  • Incidence and titer of positive anti-drug-antibodies (anti-ATYR1923) [ Time Frame: Baseline to Week 24 ]
  • Incidence and titer of anti-Jo-1 antibodies [ Time Frame: Baseline to Week 24 ]
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 Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis
Brief Summary This randomized, double-blind, placebo-controlled, study will evaluate the safety, tolerability, immunogenicity, pharmacokinetic (PK), and preliminary efficacy of multiple ascending doses of IV ATYR1923 in patients with pulmonary sarcoidosis undergoing a protocol-guided oral corticosteroid (OCS) tapering regimen.This study will consist of 3 staggered multiple dose cohorts. Each eligible participant will participate in only one cohort during the study. Within each cohort, 12 participants will be randomized 2:1 to ATYR1923 (N=8) or placebo (N=4).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
36 participants with pulmonary sarcoidosis will be randomized into one of 3 sequential cohorts, each comprising 12 participants allocated 2:1 to ATYR1923:Placebo
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Sarcoidosis
Intervention  ICMJE
  • Biological: ATYR1923 1.0 mg/kg or placebo
    ATYR1923 participants to receive ATYR1923 1.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
  • Biological: ATYR1923 3.0 mg/kg or placebo
    ATYR1923 participants to receive ATYR1923 3.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
  • Biological: ATYR1923 5.0 mg/kg or placebo
    ATYR1923 participants to receive ATYR1923 5.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
Study Arms  ICMJE
  • Experimental: Cohort 1
    ATYR1923 1.0 mg/kg or placebo
    Intervention: Biological: ATYR1923 1.0 mg/kg or placebo
  • Experimental: Cohort 2
    ATYR1923 3.0 mg/kg or placebo
    Intervention: Biological: ATYR1923 3.0 mg/kg or placebo
  • Experimental: Cohort 3
    ATYR1923 5.0 mg/kg or placebo
    Intervention: Biological: ATYR1923 5.0 mg/kg or 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 Completed
Actual Enrollment  ICMJE
 (submitted: February 17, 2021)
37
Original Estimated Enrollment  ICMJE
 (submitted: January 29, 2019)
36
Actual Study Completion Date  ICMJE June 29, 2021
Actual Primary Completion Date June 29, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Diagnosis of pulmonary sarcoidosis for ≥6 months (cutaneous and ocular involvement allowed), defined as:

    • Histologically proven diagnosis of sarcoidosis by bronchoscopy, biopsy (any organ) or bronchioalveolar lavage
    • Parenchymal lung involvement by historical radiological evidence
  • Must have symptomatic and/or active pulmonary sarcoidosis as evidence by:

    • Modified Medical Research Council Dyspnea Scale grade of >= 1; and
    • Forced vital capacity ≥50%; and
  • Receiving treatment with 10 to 25 mg/day of oral prednisone (or equivalent), at a stable dose for ≥4 weeks prior to Day 1, and capable of undergoing the protocol-specified steroid taper regimen.
  • Body weight ≥45 kg and <160 kg.

Key Exclusion Criteria:

  • Current disease presentation consistent with Lofgren's syndrome.
  • History of severe allergic or anaphylactic reactions to therapeutic proteins or known sensitivity to ATYR1923 or to its inactive components (L-histidine, sodium chloride, sucrose, L-methionine, and polysorbate-20).
  • Treatment with biological immunomodulators such as tumor necrosis factor-alpha inhibitors.
  • Current evidence of clinically significant cardiovascular, hepatic, renal, hematological, metabolic, or gastrointestinal disease, or has a condition that requires other treatment.
  • Clinically significant pulmonary hypertension requiring vasodilator treatment.
  • Any history of tuberculosis or evidence of active systemic non-tuberculosis fungal or mycobacterial infection within 1 year of Screening.
  • History of clinically significant cardiac, neurological, gastrointestinal, and/or renal manifestations of their sarcoidosis.
  • Any condition that necessitated hospitalization within the 3 months prior to Day 1 or is likely to require so during the study.
  • Participation in another clinical study of an investigational agent or device within 3 months (small molecules) / 6 months (biologics) or 5 half-lives (if known) of the agent, whichever is longer.
  • History of or positive results of screening for hepatitis B, hepatitis C or human immunodeficiency virus.
  • Is an active, heavy smoker of tobacco/nicotine-containing products (defined as >20 cigarettes/day or e-cigarette equivalent).
  • Active substance abuse or history of substance abuse within the 12 months prior to Screening.
  • Patient has received a live vaccination within 8 weeks before Day 1 or inoculation with a live vaccine is planned during study participation.
  • Positive for Jo-1 antibodies (Ab) at Screening, or past history of Jo-1 Ab positivity.
  • Significant and/or acute illness within 5 days prior to drug administration that may impact safety assessments, in the opinion of the Investigator.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03824392
Other Study ID Numbers  ICMJE ATYR1923-C-002
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
Responsible Party aTyr Pharma, Inc.
Study Sponsor  ICMJE aTyr Pharma, Inc.
Collaborators  ICMJE Foundation for Sarcoidosis Research
Investigators  ICMJE
Study Director: Gennyne Walker aTyr Pharma, Inc.
PRS Account aTyr Pharma, Inc.
Verification Date July 2021

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