NL-OMON55922已完成2 期
A Phase 2, Randomized, Double-blind, Placebo-controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease - DCR-A1AT-201
Dicerna Pharmaceuticals, Inc., a wholly owned subsidiary of Novo Nordisk0 个研究点目标入组 3 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Age 18 to 75 years, inclusive, at the time of signing the informed consent
- •form (ICF).
- •2. Documented diagnosis of PiZZ-type AATD, confirmed by genotyping. Historical
- •genotyping data may be used, if available.
- •3. AATLD, with a liver fibrosis score categorized as F1, F2, F3, or F4 in the
- •METAVIR scoring system, documented by liver biopsy during Screening.
- •4. Post-bronchodilator FEV1> 45% of predicted at Screening
- •5. Participants receiving augmentation therapy on a regular basis and intending
- •to continue augmentation therapy during the study are eligible to participate.
- •6. Estimated glomerular filtration rate at Screening >= 60 mL/min/1.73
- •7. Non-smokers (defined as having not smoked cigarettes daily for at least the
- •preceding12 months) with current non-smoking status confirmed by urine cotinine
- •at Screening AND any previous smoking history prior to 12 months must be < 15
- •pack years, including use of e-cigarettes. Participants may be on nicotine
- •replacement (patch or gum). A positive urine cotinine result due to nicotine
- •replacement is acceptable for enrollment at the discretion of the Investigator.
- •8. Male or female:
- •- Male: A male participant with a partner of childbearing potential must agree
- •to use contraception, as detailed in Section 10.4.2 of the Protocol, during the
- •treatment period and for at least 12 weeks after the last dose of study
- •intervention and refrain from donating sperm during this period. Contraceptive
- •use should be consistent with local regulations regarding the methods of
- •contraception for those participating in clinical studies.
- •- Female: A female participant is eligible to participate if she is not
- •pregnant and not breastfeeding. Women of childbearing potential (WOCBP) must be
- •using a highly effective method of contraception, as defined in Section 10.4.1.
- •of the Protocol.
- •9. Capable of giving signed informed consent, which includes compliance with
- •the requirements (including consent to undergo paired liver biopsies) and
- •restrictions listed in the ICF and in the protocol.
排除标准
- •Medical Conditions
- •1. Any condition which, in the investigator's opinion might jeopardize
- •participant's safety or compliance with the protocol.
- •2. History of chronic liver disease other than non-alcoholic fatty liver
- •disease from any cause other than PiZZ-type AATD.
- •3. Child-Pugh Score B or C
- •4.History of one single severe exacerbation of underlying lung disease in the
- •past year prior to randomization. A severe exacerbation is defined as an
- •exacerbation that requires hospitalization or a visit to the emergency room.
- •5. History of rapid decline in pulmonary function, as assessed by the
- •Investigator.
- •6. Known or suspected abuse of drugs in the opinion of the Investigator.
- •7. Known or suspected excessive consumption of alcohol (>= 21 units of alcohol
- •per week in men and >= 14 units of alcohol per week in women; where a unit of
- •alcohol is equivalent to a 12-ounce beer, 4-ounce glass of wine, or 1 ounce
- •shot of hard liquor as defined by the World Health Organization)
- •8. Any of the following: myocardial infarction, stroke, classification of heart
- •failure New York Heart Association (NYHA) Class IV, hospitalization for
- •unstable angina pectoris or transient ischaemic attack within the past 90 days
- •prior to the day of screening (V2A) and between screening and randomization.
- •9. History of malignancy, unless the malignancy (other than hepatocellular or
- •lung cancer) has been in complete remission off chemotherapy and without
- •additional medical or surgical interventions within the preceding 5 years, or
- •unless the malignancy has been an adequately treated skin cancer (other than
- •melanoma) or, superficial bladder tumor, or in situ cervical cancer in the
- •preceding 1 year.
- •Prior/Concomitant Therapy
- •10. Use of an RNAi drug at any time.
- •11. History of one or more of the following reactions to an
- •oligonucleotide-based therapy:
- •a. severe thrombocytopenia (platelet count < 100,000/ mm3)
- •b. hepatotoxicity, defined as alanine aminotransferase (ALT) or aspartate
- •aminotransferase (AST) > 3 × upper limit of normal (ULN) and total bilirubin >
- •2 × ULN or INR > 1.5
- •c. severe flu-like symptoms leading to discontinuation of therapy
- •d. localized skin reaction from the injection (graded severe) leading to
- •discontinuation of therapy
- •e. coagulopathy/clinically significant prolongation of clotting time
- •Prior/Concurrent Clinical Study Experience
- •12. Participation in any clinical study in which they received an IMP within 4
- •months (or 5 times the half-life, whichever is longer) before Screening
- •Diagnostic assessments
- •13. AST and ALT > 5 × ULN at Screening For individuals with any serum
- •aminotransferase elevation > 2 × ULN, autoimmune hepatitis should be ruled out
- •through the appropriate screening tests, which may include total IgG or
- •gamma-globulin levels and/or serologic markers (antinuclear antibodies,
- •anti-smooth-muscle antibodies at a titer of at least 1:40, anti-liver/kidney
- •microsomal-1 antibodies, anti-liver cytosol antibody [anti-LC 1], or
- •antisoluble liver/liver pancreas [anti-SLA/LP] antibodies).
- •14. alkaline phosphatase (ALP) 2 × ULN at Screening
- 另有 4 项未显示
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