NCT03925194招募中2 期
A Phase IIa, Randomized, Placebo-controlled, Double-blind, Cross-over Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients with Cystic Fibrosis
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 52
- 试验地点
- 3
- 主要终点
- Absolute pre-post change of the lung clearance index (LCI)
研究概览
简要总结
OBJECTIVES
Primary:
To evaluate efficacy of treatment with anakinra in subjects with CF who are ≥ 12 years of age by means of lung clearance index (LCI).
Secondary To evaluate safety and tolerability of treatment with anakinra as well as to investigate further effects of anakinra on lung function and quality of life (QOL) in subjects with CF.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years (1st cohort). If justified by interim analysis, 18 > age ≥ 12 years (2nd cohort),
- •Informed consent of the patient (if applicable) and/or all legal guardians,
- •Sufficient fluency of patient and/or his/her representative in German language to comply with study-specific procedures (e.g. to complete required quality of life questionnaires),
- •Confirmed diagnosis of cystic fibrosis, fulfilling at least one of the following three criteria:
- •sweat chloride ≥ 60mEq/L,
- •two CF causing mutations in the CFTR gene,
- •alterations of transepithelial potential difference of nasal or rectal epithelia typical for CF,
- •FEV1 ≥ 50 % pred. at screening,
- •LCI2.5 ≥ 7.05 at screening,
- •Ability to perform reproducible multiple breath washout and spirometry,
- •Oxyhaemoglobin saturation of ≥ 90% on room air at screening,
- •No changes in the medication for cystic fibrosis lung disease for at least 4 weeks prior to the first administration of the IMP of each treatment period (in case of medication changes in Period 1 and/or the washout phase the wash-out may be extended for up to 12 weeks in order to fulfill this criterion),
- •Adequate bone marrow function assessed on the basis of: neutrophils >1.5 x 109/L, platelets >100 x 109/L, hemoglobin >9.0 g/dL,
- •Adequate liver function assessed on the basis of: GGT, ASAT, and ALAT <3 x upper limit of normal (ULN),
- •Adequate blood clotting assessed on the basis of: aPTT <39 sec., INR <1.2,
- •Negative serology for HIV (anti-HIV 1/2 IgG/IgM and p24-Ag), HBV (anti-HBs quantitative and anti-HBc IgG/IgM) and HCV (anti-HCV IgG), negative Interferon-gamma release assay,
- •Negative Beta-HCG blood/urine test in women of childbearing potential (of childbearing potential are females who have experienced menarche and are not permanently sterile or postmenopausal (postmenopausal: 12 consecutive months with no menses without an alternative medical cause)),
- •Use of adequate contraception in sexually active female subjects (sexual abstinence, hormonal contraceptives or intrauterine device).
排除标准
- •Expected non-compliance, i.e. inability or unwillingness to comply with study-specific procedures,
- •Known allergy to anakinra or any ingredient of the pharmaceutical formulation of Kineret®,
- •Planned immunization with attenuated (live) vaccine(s) during the treatment with the IMP or completed immunization with attenuated (live) vaccine(s) within 4 weeks prior to the first administration of the IMP,
- •Renal failure (creatinine in serum above ULN),
- •History of tuberculosis or repeated detection of non-tuberculous mycobacteria from airway samples in the last 12 months before start of each treatment period,
- •History of detection of Burkholderia cenocepacia species in the last 12 months before start of each treatment period,
- •Colonization with multi-resistant Staphylococcus aureus (MRSA) and/or 4-multi-resistant gram negative (MRGN) Pseudomonas aeruginosa is only an exclusion criterion if the treating physician judges that this is an increased risk for the patient,
- •Acute bronchopulmonary exacerbation (defined by modified Fuchs criteria (1) (see Appendix 1), modification includes all ways of application of an antibiotic (e.g., oral, i.v., inhaled)) within 14 days prior to the screening and before start of each treatment period,
- •Signs of other active infection within 14 days prior to the screening and before start of each treatment period (clinical symptoms (e.g. burning sensation while urinating, skin, wound or dental infection) and/or fever and/or deterioration of infection-specific laboratory parameters beyond changes driven by the underlying disease),
- •Immunosuppressive treatment due to organ transplantation, rheumatic or autoimmune diseases as well as treatment with Anakinra in the last 3 months before Day 1 of Period 1,
- •Participation in another interventional trial within the last 30 days prior to screening,
- •Current oral corticosteroid use,
- •Current oxygen supplementation,
- •Current treatment with etanercept,
- •Medical history of lung transplantation,
- •Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening),
- •Known hypersensitivity to hypertonic saline (used for induction of sputum).
研究组 & 干预措施
Anakinra
Experimental
干预措施: Anakinra (Drug)
Placebo
Placebo Comparator
干预措施: Anakinra (Drug)
结局指标
主要结局
Absolute pre-post change of the lung clearance index (LCI)
时间窗: 28 days
次要结局
未报告次要终点
研究者
Mirjam Stahl
Deputy of Principal Investigator (Olaf Sommerburg)
Heidelberg University
研究点 (3)
Loading locations...
相似试验
已完成
3 期
Ferinject® Assessment in Patients With Iron Deficiency and Chronic Heart Failure (FAIR-HF)Iron DeficiencyAnaemiaIron Deficiency AnemiaChronic Heart FailureNCT00520780Vifor Pharma456
已完成
2 期
First Line Metastatic Colorectal Cancer Therapy in Combination With FOLFOXColorectal CancerNCT00384176AstraZeneca1,814
已完成
1 期
Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR MutationCystic FibrosisNCT02275936Nivalis Therapeutics, Inc.51
Unknown
2 期
Tyrosine Kinase Inhibitor (TKI) + Anti-PD-1 Antibody in TKI-responded Microsatellite Stability/Proficient Mismatch Repair (MSS/pMMR) Metastatic Colorectal Adenocarcinoma.MSSpMMRMetastatic Colorectal AdenocarcinomaNCT04483219China Medical University, China53
撤回
2 期
Phase II Study of Fludarabine, Cytarabine (ARA-C) and Erwinase IV in Patients With Relapsed or Refractory Hematologic MalignanciesHematologic MalignancyNCT02718755M.D. Anderson Cancer Center