Phase III Study to Evaluate Clinical Efficacy and Safety of Avian Polyclonal Anti-Pseudomonas Antibodies (IgY) in Prevention of Recurrence of Pseudomonas Aeruginosa Infection in Cystic Fibrosis Patients
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 164
- 试验地点
- 42
- 主要终点
- Time from start of treatment (=Day 0) to the first recurrence of PA (Pseudomonas aeruginosa) in the sputum or throat cough swab or endolaryngeal suction
研究概览
简要总结
The purpose of this study is to prolong the time to reinfection with Pseudomonas aeruginosa after successfully treated acute or intermittent infection.
详细描述
This is a double -blind, placebo controlled study in which the investigational drug and the reference placebo group are gargled and swallowed. 70 ml IgY/ placebo solution is gargled every night for two minutes (for maximal 24 months) The design will include the recruitment of 144 patients randomized in two groups (72 per treatment group) In order to compensate for dropouts (i.e. patients dropping out prior to 24 months without having an event) the total sample size was planned to be approximately 180 (i.e. ~20 % dropout rate). After the actual drop-out rate has been low throughout the study, only 144 plus approx. 10% potential drop-outs were included into the study.
During the two years of treatment, subjects will be examined at the clinic every 3 months regarding safety and efficacy of the medication.
For more information please see www.impactt.eu The IMPACTT Project is funded by EU within the Framework 7 Program. PsAer-IgY Studies is part of IMPACTT Project (Workpackage 2).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •CF patients diagnosed according to specific clinical features and either a positive sweat chloride in double proofs or presence of disease-associated CFTR mutations in both alleles
- •Males and females 5 years of age and above (being able to gargle)
- •CF patients having a FEV1 value between 50% and 130% of predicted value (according to Knudson formula)
- •CF patients who have had one to several sputum or throat cough swabs or endolaryngeal suction cultures positive for PA within the last three years and for whom PA has been successfully eradicated.
- •Sputum / throat cough swab/ endolaryngeal suction culture negative for PA and other gram-negative bacteria on study entry.
- •Patients and/ or their legal representative who are willing and able to give informed consent/ assent to participate in the study after thorough information
- •Subjects of child bearing potential and who are sexually active must meet the contraception requirements (i.e. oral or injectable contraceptives, intrauterine devices, double-barrier method, contraceptive patch, male partner sterilization or condoms).
排除标准
- •Microbiologic or serologic evidence of chronic infection with PA. Definition of chronic PA infection: Three cultures (sputum or throat cough swabs or endolaryngeal suction) have been positive for PA for 6 consecutive months (at least 3 cultures have to be taken) or more, .
- •Patients, who have positive sputum culture or throat cough swab or endolaryngeal suction culture for gram-negative bacteria, such as PA, S. maltophilia, B. cepacia, A. xylosoxidans (eradication before entry in study is possible), Patients, who have positive sputum culture or throat cough swab or endolaryngeal suction culture for atypical Mycobacteria and / or Aspergillus fumigates, associated with clinical symptoms that may necessitate specific treatment.
- •History of allergy/hypersensitivity to hens' egg proteins (including medication allergy) that is deemed relevant to the trial by the investigator. "Relevance" in this context refers to any increased risk of hypersensitivity reaction to trial medication.
- •Patient with a known relevant substance abuse, including alcohol or drug abuse.
- •Start of a new concomitant or chronic medication for CF within 4 weeks before inclusion.
- •Clinically relevant diseases or medical conditions other than CF or CF-related conditions that, in the opinion of the investigator, would compromise the safety of the patient or the quality of the data. This includes, but is not limited to, significant hematological, hepatic, renal, cardiovascular, and neurological diseases (diabetic patients may participate if their disease is under good control prior to inclusion).
- •Participation in another study with an investigational drug within one month or 6 half-lives (whichever is greater) preceding the inclusion.
- •The patient is an employee of the investigator or the institution with direct involvement in the trial or other trials under the direction of the investigator or their members.
- •Patients who are pregnant cannot be included into the study. This will be tested at inclusion visit with a urine pregnancy test (in female patients older than 10 years with secondary sexual characteristics)
研究组 & 干预措施
IgY, gargling solution
Avian polyclonal anti-pseudomonas antibodies (IgY), 70 ml gargling solution contains 50 mg IgY with an activity against PA, once daily
干预措施: IgY (Drug)
Placebo, gargling solution
70 ml gargling solution without antibodies, once daily
干预措施: Placebo (Drug)
结局指标
主要结局
Time from start of treatment (=Day 0) to the first recurrence of PA (Pseudomonas aeruginosa) in the sputum or throat cough swab or endolaryngeal suction
时间窗: max. 24 months
次要结局
- • Change in FEV 1.0 from day 0 to each visit(max. 24 months)
- • Change in BMI from day 0 to each visit(max. 24 months)
- • Number of exacerbations(max. 24 months)
- • Number of days of illness in hospital and at home, i.e. out of school or work(max. 24 months)
- • Control of use of antibiotics, especially anti-pseudomonas antibiotics -measured as days with antibiotic treatment(max. 24 months)
- • Change in values of serologic tests for PA precipitins from day 0 to each visit (if applicable)(max. 24 months)
- • Good tolerability and comparable number and quality of adverse events like placebo group(max. 24 months)
- • Sputum or throat cough swab or endolaryngeal suction cultures for bacteria and fungi(max. 24 months)