EUCTR2008-005326-36-NL进行中（未招募）1 期
A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema
Kamada Limited0 个研究点目标入组 168 人开始时间: 2009年8月21日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 168
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
- •For the double-blind period:
- •1. Diagnosis of emphysema confirmed by CT scan. If a report of past CT scan is not available at site documenting diagnosis then a CT scan is to be performed at screening
- •2. Male or female patients at least 18 years of age.
- •3. Able and willing to sign an informed consent.
- •4. Patient with record of congenital AAT deficiency of phenotype PiZZ (homozygote) or other rare phenotypes related to AAT deficiency and with AAT serum level lower or equivalent to 11 micromole. For patients receiving IV AAT augmentation therapy the serum AAT level threshold does not apply.
- •5. FEV1/SVC <70% of predicted value post bronchodilator (SVC is slow VC) and FEV1 < 80% of predicted value post-bronchodilator
- •6. History of at least two moderate or severe exacerbations that required change in treatment (. antibiotics, systemic steroids, hospitalization) in the last 18 months prior to date of screening, with at least one of these occurring within the last 12 months prior to screening.
- •7. Ability to comply with completion of electronic diary.
- •8. Ability to self-administer inhaled AAT using the eFlow device.
- •9. No significant abnormalities in serum hematology, serum chemistry and serum inflammatory / immunogenic markers according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.
- •10. No significant abnormalities in urinalysis according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.
- •11. No significant abnormalities in ECG per investigator judgment.
- •12. Negative for HBsAg and antibodies to HCV, HIV-1.
- •13. AAT deficient patients who are either naïve (not receiving IV augmentation therapy) or AAT deficient patients (receiving IV augmentation therapy), if they have been stable on regular therapy for at least 3 months prior to the screening visit and are willing to continue the same regime throughout this trial. Note that only sites in Germany can recruit patients who are currently being treated with IV-AAT. Patients who stopped IV augmentation treatment 6 months prior to screening date and will not re-start this treatment for the course of the study will be considered Naïve.
- •14. Non-pregnant, non-lactating female patients, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator, or who are at least 2 years post-menopausal or surgically sterilized.
- •For open-label extension period:
- •1. Patients who have completed the 50-week treatment period in the double-blind study as per the protocol.
- •2. Patients who have already terminated their participation in the double-blind period prior to the time of implementation of the OL extension period in the protocol, provided that the patient:
- •a. received study treatment for at least 6 months in the double-blind study
- •b. did not prematurely discontinue from the study due to lost to follow-up or non-compliance with study requirements.
- •3. For women of childbearing potential, negative pregnancy test and use of contraceptive methods deemed reliable by the investigator. Women are not considered to be of childbearing potential if they are at least 2 years postmenopausal or surgically sterilized
- •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
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Nu
排除标准
- •For the double-blind period:
- •1. FEV1 = 80% or FEV1 < 20% of predicted value post-bronchodilator.
- •2. FEV1/SVC=70%
- •3. History of lung transplant.
- •4. Any lung surgery within the past two years.
- •5. On any thoracic surgery waiting list.
- •6. End of last exacerbation less than 6 weeks prior to screening/re-screening visit.
- •7. Clinically significant intercurrent illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal or other) that in the opinion of the investigator, could interfere with the safety, compliance or other aspects of this study. Patients with well-controlled, chronic diseases could be possibly included after consultation with the treating physician and the sponsor.
- •8. Active smoking during the last 12 months from screening date.
- •9. Pregnancy or lactation.
- •10. Woman of child-bearing potential not taking adequate contraception deemed reliable by the investigator.
- •11. Presence of psychiatric/ mental disorder or any other medical disorder which might impair the patient’s ability to give informed consent or to comply with the requirements of the study protocol.
- •12. Evidence of ongoing viral infection with HCV, HBV and/or HIV.
- •13. Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally prescribed drugs.
- •14. IgA Deficiency
- •15. History of life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
- •16. Participation in another clinical trial within 30 days prior to baseline visit.
- •17. Inability to attend scheduled clinic visits and/or comply with the study protocol.
- •18. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.
- •For the open-label extension period:
- •1. Clinically significant adverse event(s) in the double-blind period or clinically significant intercurrent illness after termination of participation in the doubleblind period that, in the opinion of the investigator, might prevent the patient from safely participating in the OL extension period of the study.
- •2. A clinically significant deterioration in lung function that, in the opinion in the investigator, might prevent the patient from safely participating in the OL extension period of the study.
- •3. Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally prescribed drugs.
- •4. Occurrence of a life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
- •5. Received lung transplant or underwent lung surgery since termination of participation in the double blind study.
- •6. Active smoking.
- •7. Pregnancy or lactation.
- •8. Women of childbearing potential not taking adequate contraception deemed reliable by the investigator.
- •9. Participation in another clinical trial since termination of participation in the double-blind period of the study.
- •10. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol or would jeopardize the safety of the patient.
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