2023-506039-13-00招募中3 期
A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRAVENOUS EFZOFITIMOD IN PATIENTS WITH PULMONARY SARCOIDOSIS
aTYR Pharma Inc.27 个研究点 分布在 5 个国家目标入组 101 人开始时间: 2023年7月10日最近更新:
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 101
- 试验地点
- 27
- 主要终点
- Change from baseline in mean daily OCS dose at Week 48
研究概览
简要总结
To assess the efficacy of efzofitimod in patients with pulmonary sarcoidosis
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Male or females aged 18 to 75 years, inclusive at the time of informed consent
- •Confirmed diagnosis of pulmonary sarcoidosis for at least 6 months, defined by the following criteria: 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: a. Modified Medical Research Council (MRC) Dyspnea Scale grade of at least 1 b. KSQ-Lung score ≤70
- •Patients must be receiving treatment with OCS of ≥ 3 months with a starting dose between ≥ 7.5 and ≤ 25 mg/day.
- •Body weight ≥ 40 kg and < 160 kg
- •If female of childbearing potential, must a. Not be pregnant or lactating, and have a negative pregnancy test at Screening (serum) and at Day 1 (urine) prior to first study drug infusion b. Be willing to use acceptable or highly effective methods of contraception from Screening until 8 weeks after the last study drug administration (refer to Appendix 6 for acceptable and highly effective methods of contraception) Note: To be considered of non-childbearing potential, the patient must be either surgically sterile or postmenopausal (confirmed by amenorrhea duration of at least 12 months with no alternative medical cause
- •Provide written informed consent
- •Agree to comply with all study procedures and requirements
排除标准
- •Current disease presentation consistent with Lofgren’s syndrome (ie, presence of the triad of erythema nodosum, bilateral hilar lymphadenopathy on chest X-ray, and joint pain
- •History of severe allergic or anaphylactic reactions to therapeutic proteins or known sensitivity to efzofitimod or its inactive components (L-histidine, sodium chloride, sucrose, L-methionine, and polysorbate-20)
- •In the opinion of the Investigator and Medical Monitor, current evidence of clinically significant cardiovascular, hepatic, neurological, renal, hematological, lymphatic, metabolic, or gastrointestinal disease, or any condition that requires other treatment or surgery, that may preclude the assessment of efficacy, confound the assessment of safety, or compromise patients’ compliance with study procedures
- •Active or history of malignancy within the last 5 years, except for resected basal cell carcinoma, squamous cell carcinoma of the skin, or effectively managed cervical carcinoma
- •Major surgery or hospitalization within 3 months prior to Day 1 or anticipated surgery during the study
- •Participation in another clinical study of an investigational agent or device within 3 months (small molecules and device), 6 months (biologics), or 5 half-lives (if known) of the agent, whichever is longer
- •Is an active, heavy smoker of tobacco/nicotine-containing products
- •Active substance abuse (drugs, alcohol, or cannabis) or history of substance abuse within 12 months prior to Screening
- •Clinically significant abnormalities in the Screening physical examination, vital signs, ECG, or clinical laboratory test results that, in the opinion of the Investigator and Medical Monitor, preclude the patient’s participation in the clinical study
- •History of anti-synthetase syndrome or Jo-1 positive at baseline
- •Treatment with > 1 oral 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: a. CT fibrosis > 20% within the last 12 months b. FVC percent predicted (FVCPP) < 50% c. KSQ-Lung score < 30
- •Clinically significant bronchiectasis or cavitary sarcoidosis with mycetoma at Screening or during the previous 12 months
- •Clinically significant pulmonary hypertension requiring treatment with vasodilators
- •Patients with cardiac sarcoidosis, neurosarcoidosis, or renal sarcoidosis
- •Clinically significant cutaneous and ocular sarcoidosis
- •History of Addisonian symptoms that precluded previous OCS taper attempts
研究组 & 干预措施
-
Placebo Comparator
Participants receiving -
干预措施: - (Drug)
结局指标
主要结局
Change from baseline in mean daily OCS dose at Week 48
Change from baseline in mean daily OCS dose at Week 48
次要结局
- 1. Steroid-free rate at Week 48
- 2. Steroid-free and stable KSQ-Lung composite rate at Week 48
- 3. Change from baseline in absolute value of FVC at Week 48
研究者
Regulatory Submissions
Scientific
aTYR Pharma Inc.
研究点 (27)
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