Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03824392
Recruitment Status : Recruiting
First Posted : January 31, 2019
Last Update Posted : March 15, 2019
Sponsor:
Collaborator:
Foundation for Sarcoidosis Research
Information provided by (Responsible Party):
aTyr Pharma, Inc.

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).

Condition or disease Intervention/treatment Phase
Pulmonary Sarcoidosis Biological: ATYR1923 1.0 mg/kg or placebo Biological: ATYR1923 3.0 mg/kg or placebo Biological: ATYR1923 5.0 mg/kg or placebo Phase 1 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
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
Official Title: A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis
Actual Study Start Date : January 29, 2019
Estimated Primary Completion Date : June 21, 2020
Estimated Study Completion Date : June 21, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Sarcoidosis

Arm Intervention/treatment
Experimental: Cohort 1
ATYR1923 1.0 mg/kg or placebo
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

Experimental: Cohort 2
ATYR1923 3.0 mg/kg or placebo
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

Experimental: Cohort 3
ATYR1923 5.0 mg/kg or placebo
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




Primary Outcome Measures :
  1. Incidence of treatment emergent adverse events and serious adverse events [ Time Frame: Baseline to Week 24 ]

Secondary Outcome Measures :
  1. Total cumulative steroid dose administered over study period [ Time Frame: Baseline to Week 24 ]
  2. Number of participants who achieve and maintain the targeted tapered dose of prednisone 5 mg/kg day [ Time Frame: Baseline to Week 24 ]
  3. Exposure response analysis comparing steroid dose area under the curve (AUC) with ATYR1923 PK parameters [ Time Frame: Baseline to Week 24 ]
  4. Incidence and titer of positive anti-drug-antibodies (anti-ATYR1923) [ Time Frame: Baseline to Week 24 ]
  5. Incidence and titer of anti-Jo-1 antibodies [ Time Frame: Baseline to Week 24 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Diagnosis of pulmonary sarcoidosis for ≥1 year (cutaneous and ocular involvement allowed), defined as:

    • Histologically proven diagnosis of sarcoidosis by biopsy 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 ≤90 percent predicted; and
    • Positive 18F-fluorodeoxyglucose positron-emission tomography/computed tomography (18F-FDG-PET/CT) scan showing increased metabolic activity in lung parenchyma within 4 weeks prior to Day 1.
  • 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 ≥55 kg and <140 kg.

Key Exclusion Criteria:

  • Current disease presentation consistent with Lofgren's syndrome.
  • History of severe allergic or anaphylactic reactions to therapeutic proteins.
  • 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.
  • History or evidence of active or latent systemic fungal or mycobacterial infection.
  • 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.
  • Has smoked or inhaled (including e-cigarettes or e-vaporizers) any nicotine containing product within 6 months prior to Screening.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03824392


Contacts
Layout table for location contacts
Contact: aTyr Pharma Clinical Research 877-215-5731 clinicaltrials@atyrpharma.com

Locations
Layout table for location information
United States, Colorado
National Jewish Health Recruiting
Denver, Colorado, United States, 80206
Contact    303-398-1708    egidioc@njhealth.org   
Principal Investigator: Lisa Maier, MD         
United States, New York
Albany Medical Center Recruiting
Albany, New York, United States, 12208
United States, Ohio
University of Cincinnati Recruiting
Cincinnati, Ohio, United States, 45267
Contact: Rebecca Reeves, RN, BSN    513-584-5226    reevessrc@ucmail.uc.edu   
Principal Investigator: Robert Baughman, MD         
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 92425
Contact: Brooke Alexander, CRC    843-792-1221    pulmonaryresearch@musc.edu   
Principal Investigator: Ennis James, MD         
Sponsors and Collaborators
aTyr Pharma, Inc.
Foundation for Sarcoidosis Research
Investigators
Layout table for investigator information
Study Director: Gennyne Walker aTyr Pharma, Inc.

Layout table for additonal information
Responsible Party: aTyr Pharma, Inc.
ClinicalTrials.gov Identifier: NCT03824392     History of Changes
Other Study ID Numbers: ATYR1923-C-002
First Posted: January 31, 2019    Key Record Dates
Last Update Posted: March 15, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by aTyr Pharma, Inc.:
Pulmonary Sarcoidosis
Resokine
Sarcoidosis
Granulomas
Inflammation
Lymphoproliferative Disorders
Interstitial Lung Disease
Neuropilin-2
Steroids
Oral corticosteroids
Immunomodulatory
tRNA Synthetase
ATYR1923

Additional relevant MeSH terms:
Layout table for MeSH terms
Sarcoidosis
Sarcoidosis, Pulmonary
Lymphoproliferative Disorders
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases