2025-523497-16-00招募中2 期
TSRA196-AAT-201: A Phase 1/2, Open-Label, Multi-Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA-196 in Adults With the PiZZ Genotype Who Have Lung and/or Liver Disease Associated with Severe Alpha-1 Antitrypsin Deficiency
Tessera Therapeutics Inc.5 个研究点 分布在 3 个国家目标入组 25 人开始时间: 2026年4月30日最近更新:
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 25
- 试验地点
- 5
研究概览
简要总结
Part 1: To evaluate the safety and tolerability of a single dose of TSRA-196 in adults with AATD Part 2: To evaluate the efficacy of a single dose of TSRA-196 in adults with AATD Part 3: To evaluate the efficacy of a second dose of TSRA-196 in adult participants with AATD
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
- •Males or females who are 18 to 70 years of age, inclusive, at the time of signing the informed consent
- •Part 1A and 2A (AATD lung disease with no or minimal liver fibrosis) • Clinically significant lung disease at baseline that is not more severe than specified in the exclusion criteria • No or minimal liver fibrosis determined by liver biopsy or non-invasive assessment at baseline
- •Part 1B and 2B (AATD liver disease with significant or severe liver fibrosis, with or without AATD lung disease) • Moderate-to-severe liver fibrosis determined by liver biopsy at baseline
- •Body mass index of 18 to 37 kg/m2, inclusive
- •Confirmed diagnosis of AATD and PiZZ genotype
- •At least one previous measure of blood total AAT level <11 μmol/L
- •Nonsmoker for at least 6 months before screening and must remain nonsmoking for the entire study duration
- •Agrees to not consume alcohol for 14 days before TSRA-196 administration and for 30 days after TSRA-196 administration and understands that they should not consume significant amounts of alcohol consumption for the entire study duration.
- •Total bilirubin value at screening with or without Gilbert’s syndrome as defined in the protocol.
- •Laboratory, hematology values as defined in the protocol
- •Either AAT treatment-naïve or washed out of all investigational or approved treatments that modify AAT levels
排除标准
- •Presence of genetic variation in the region around the PiZ variant that may disrupt the function of TSRA-196, determined by screening genotyping.
- •Significant lung disease not attributable to manifestations of AATD, as determined by the investigator.
- •History of one or more hospitalizations due to severe exacerbation of underlying lung disease during the year before screening or received IV antibiotics for treatment of a pulmonary infection within 6 months before screening.
- •Unstable AATD-related COPD, as determined by the investigator, or severe bronchiectasis
- •Lung volume reduction surgery within 1 year before screening or plan to receive lung volume reduction surgery during the study period (between Screening and the Month 12 EOS visit).
- •Documented chronic need for positive airway pressure therapy beyond nocturnal use
- •Has received an organ transplant or is on a waiting list for an organ transplant.
- •Dependent on continuous supplementary oxygen
- •Immunization within 30 days before dosing for attenuated live vaccines and 14 days before dosing for all other vaccines.
- •Prior treatment with gene therapy using viral vectors or intended to permanently change the patient’s DNA. Prior treatment with RNA therapeutics is acceptable.
- •Seropositive for HIV (HIV-1 or HIV-2), active hepatitis B (HBsAg or HBcAb positive with detectable HBV DNA), or hepatitis C infection (Parts 1A/2A: HCV RNA positive; Parts 1B/2B: HCV RNA and/or antibody positive)
- •History of thromboembolic disease, myocardial infarction, or stroke within 6 months of screening
- •Uncontrolled hypertension as defined in the protocol
- •History of LNP-related reaction classified as CTCAE Grade 3 or higher
- •History of active malignancy within 5 years before screening, except basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, cervical carcinoma in situ curatively treated or low-grade prostate adenocarcinoma for which appropriate management is observation alone.
- •Presence of a null or projected loss-of-function variant in the SERPINA1 gene determined by screening genotyping.
- •History of liver disease unrelated to AATD
- •History of or clinical signs of cirrhosis, e.g., varices or ascites.
研究组 & 干预措施
TSRA-196
Test
干预措施: TSRA-196 (Drug)
研究者
Susan Sparks
Scientific
Tessera Therapeutics Inc.
研究点 (5)
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