EUCTR2020-003313-35-ES进行中（未招募）1 期
A Phase 2a, Randomized, Double-blind, Placebo-controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients with PiZZ Alpha-1 Antitrypsin Deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 54
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Aged 18 to 70 years, inclusive, at the time of signing the ICF
- •2. Documented diagnosis of PiZZ-type AATD, confirmed by genotyping. Historical genotyping data may be used, if available.
- •3. AATLD-associated liver disease, with a fibrosis score categorized as F2, F3, or F4 in the METAVIR scoring scale, documented by liver biopsy.
- •4. Consent to undergo paired liver biopsies, one at Screening and one at either 24 (± 2) weeks or 48 (± 2) weeks afterof the first dose of the study intervention
- •5. Pre- and Post-bronchodilator FEV1 > 60% of predicted at Screening
- •6. Postbronchodilator DLCO = 50% of predicted at Screening
- •7. Serum AAT protein concentration > 10 mg/dL at the first assessment during Screening
- •8. eGFR at Screening = 60 mL/min normalized to 1.73 m^2 BSA
- •9. Smoking cessation for at least 3 months prior to Screening
- •10. Body mass index (BMI) within the range of 18 to 35 kg/m^2 (inclusive)
- •11. 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, not breastfeeding, and not a WOCBP, as defined in Section 10.4.1. of the Protocol
- •12. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 49
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially affect patient safety including, but not restricted to:
- •a. severe intercurrent illness
- •b. physician concerns about intake of drugs of abuse, alcohol use disorder, excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as = 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)
- •c. clinically relevant history or presence of uncontrolled cardiovascular, respiratory, gastrointestinal, hematological, lymphatic, neurological, musculoskeletal, genitourinary, immunological diseases, and dermatological (including rash, severe eczema, or dermatitis) or connective tissue diseases or disorders. Diagnostic testing exclusions are defined in the Diagnostic Assessments section below
- •2. History of chronic liver disease from any cause other than PiZZ-type AATD, including hepatitis B, hepatitis C, primary biliary cirrhosis, primary sclerosing cholangitis, cholestatic liver disease, and autoimmune liver diseases. Diagnostic testing exclusions are defined in the Diagnostic Assessments section below.
- •3. Child-Pugh Score B or C
- •4. History of acute exacerbations of underlying lung disease in the 3 months prior to Screening, defined as increased dyspnea, sputum volume, and sputum purulence. Individuals meeting this criterion may be re-screened after 2 months
- •5. History of intolerance to SC injection(s) or significant abdominal scarring that could potentially hinder study intervention administration or evaluation of local tolerability
- •6. AAT augmentation therapy in the 6 months prior to Screening
- •7. Routine use of acetaminophen/paracetamol Note. Occasional use of 1000mg/day is acceptable
- •8. Use of systemically acting steroids in the month prior to Screening and throughout the study period. Occasional use is acceptable if needed for patient safety, e.g., in the case of an exacerbation of underlying pre-existing conditions.
- •9. Routine use of NSAIDs, or use within 3 days of dosing
- •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/ mm^3)
- •b. hepatotoxicity, defined as ALT or AST > 3 × 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
- •12. Participation in any clinical study in which they received an IMP within 4 months before Screening
- •13. AST and ALT > 5 × ULN at Screening Note: 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 anti-soluble liver/liver pancreas [anti-SLA/LP] antibodies).
- •14. ALP 2 × ULN at Screening
- •15. Serum alpha fetoprotein (AFP) value > 100 ng/mL at Screening If AFP at screening is > ULN but < 100 ng/mL, the participant is
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