Study of Intravenous ATYR1923 (Efzofitimod) for Pulmonary Sarcoidosis
Purpose
This randomized, double-blind, placebo matched to efzofitimod-controlled, study will evaluate the safety, tolerability, immunogenicity, pharmacokinetic (PK), and preliminary efficacy of multiple ascending doses of IV efzofitimod in participants 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 efzofitimod (N=8) or placebo matched to efzofitimod (N=4).
Condition
- Pulmonary Sarcoidosis
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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 evidenced 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.
Exclusion Criteria
- Current disease presentation consistent with Lofgren's syndrome. - History of severe allergic or anaphylactic reactions to therapeutic proteins or known sensitivity to efzofitimod 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 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. - Participant 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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- 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 efzofitimod:placebo matched to efzofitimod
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Placebo Comparator Placebo |
Participants will receive placebo matched to efzofitimod via intravenous (IV) infusion every 4 weeks until Week 20. |
|
Experimental Efzofitimod 1.0 mg/kg |
Participants will receive efzofitimod 1.0 milligrams/kilogram (mg/kg) via IV infusion every 4 weeks until Week 20. |
|
Experimental Efzofitimod 3.0 mg/kg |
Participants will receive efzofitimod 3.0 mg/kg via IV infusion every 4 weeks until Week 20. |
|
Experimental Efzofitimod 5.0 mg/kg |
Participants will receive efzofitimod 5.0 mg/kg via IV infusion every 4 weeks until Week 20. |
|
More Details
- Status
- Completed
- Sponsor
- aTyr Pharma, Inc.