Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 2 August 2021
• Main ID: NCT03824392
• Date of registration: 28/01/2019
• Prospective Registration: Yes
• Primary sponsor: aTyr Pharma, Inc.
• Public title: Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis
• Date of first enrolment: January 29, 2019
• Target sample size: 37
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03824392
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Gennyne Walker
• Affiliation: aTyr Pharma, Inc.

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Sarcoidosis

Intervention(s)

Biological: ATYR1923 3.0 mg/kg or placebo

Biological: ATYR1923 5.0 mg/kg or placebo

Biological: ATYR1923 1.0 mg/kg or placebo

Primary Outcome(s)

Incidence of treatment emergent adverse events and serious adverse events [Time Frame: Baseline to Week 24]

Secondary Outcome(s)

Incidence and titer of anti-Jo-1 antibodies [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]

Total cumulative steroid dose administered over study period [Time Frame: Baseline to Week 24]

Secondary ID(s)

ATYR1923-C-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Foundation for Sarcoidosis Research