EUCTR2019-000602-30-IE进行中（未招募）1 期
A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%), Followed by a 2-Year Open-Label Extension - Phase III, Efficacy and Safety of Kamada-AAT for Inhalation
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Kamada Ltd.
- 入组人数
- 220
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
- •Double-Blind Period
- •1. Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes confirmed by genotype blood test documented prior to screening.
- •2. Serum AAT levels = 11 µM at screening.
- •3. Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC=70%) at screening.
- •4. 40% = FEV1 = 80% of predicted post-bronchodilator at screening.
- •5. Patients who are either naïve or washed out of any AAT treatment for at least 8 weeks prior to randomization.
- •6. Age between 18 to 65 years inclusive at screening.
- •7. Able to read and sign informed consent and willing to participate in the study.
- •8. Males or non-pregnant, non-lactating females whose screening pregnancy test is negative, who are using contraceptive methods deemed reliable by the investigator, who are post-menopausal, or are surgically sterilized.
- •9. High compliance during run in defined as study medication use and e-Diary compliance for at least 20 out of the 28 days of run in.
- •10. Study medication use for at least 20 out of the 28 days of run-in, as recorded in the study nebulization PARI Track data.
- •101 Demonstrated ability to complete eDiary for at least 20 out of the first 28 days of run-in.
- •Open-Label Period
- •1. Patients who completed 104 weeks of DB study treatment and attended the end of treatment visit.
- •2. Patients who completed the DB period and attended follow-up visits are eligible for the OLE provided that they comply with all other OLE eligibility criteria.
- •3. Consenting to continue study participation in the OLE phase.
- •4. Agree to continue using contraceptive methods deemed reliable by the investigator for an additional 2 years, unless post-menopausal 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 220
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Double-Blind Period
- •1. Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels<0.05 g/L at screening.
- •2. History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.
- •3. Two or more moderate or any severe exacerbation(s) within the year prior to the baseline visit.
- •4. A moderate exacerbation within 6 weeks prior to the baseline.
- •5. Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).
- •6. Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal, or other. Patients might be included after consultation with the treating physician and the sponsor if, in the opinion of the Investigator, their condition will not interfere with the safety, compliance or other aspects of this study.
- •7. Hospitalization for any cause 6 weeks prior to screening.
- •8. History of lung or liver transplant.
- •9. On any thoracic or hepatic surgery waiting list.
- •10. Any lung surgery within the past two years (including bronchoscopic lung volume reduction).
- •11. Any smoking within the year prior to screening.
- •12. Evidence of alcohol abuse or history of alcohol abuse, or use of illegal drugs and/or abuse of legally prescribed drugs in the last 5 years prior to screening.
- •13. Acute or chronic hepatitis (hepatitis A, hepatitis B, hepatitis C,) or positive human immunodeficiency virus (HIV) serology.
- •14. Signs of significant abnormalities in serum hematology, serum chemistry, serum inflammatory / immunogenic markers and urinalysis per investigator judgment, taking into considerations the potential effects of the AAT deficiency.
- •15. Signs of significant abnormalities in ECG per investigator judgment at screening.
- •16. Presence of psychiatric/ mental disorder or any other medical disorder that might impair the patient’s ability to give informed consent or to comply with the requirements of the study protocol. If, in the opinion of the Investigator, the condition will not interfere with the compliance or other aspects of this study, the patient might be included after consultation with the treating physician and the sponsor.
- •17. Participation in another clinical trial involving investigational medication or interventional treatment within 30 days and/or last dose 5 half-lives prior to screening visit.
- •18. Inability to attend scheduled clinic visits and/or comply with study protocol.
- •19. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.
- •Open-Label Period
- •1. Any adverse event(s) in the DB period and/or medical condition that, in the opinion of the investigator, might prevent the patient from safely participating in the OLE period of the study, including but not limited to:
- •a. Occurrence of a life-threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
- •b. Received lung transplant, entered a waiting list for lung transplantation, or underwent lung surgery. The investigator should consult the sponsor before inclusion of any patient with a significant condition if the investigator believes that it will not pose an unacceptable risk for the patient.
- •2. Evidence of alcohol abuse or history of a
研究者
相似试验
进行中（未招募）
1 期
A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%), Followed by a 2-Year Open-Label Extension.Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predictedFEV1/SVC = 70%), and with no history of two or more moderate or one or more severe exacerbations of COPD during the past year.MedDRA version: 20.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-000602-30-FIKamada Ltd.220
进行中（未招募）
1 期
A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%), Followed by a 2-Year Open-Label ExtensioAdult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predictedFEV1/SVC = 70%), and with no history of two or more moderate or one or more severe exacerbations of COPD during the past year.MedDRA version: 20.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-000602-30-SEKamada Ltd.220
招募中
3 期
A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of *Kamada-AAT for Inhalation* 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% <= FEV1 <= 80% of predicted; FEV1/SVC <= 70%), Followed by a 2-Year Open-Label Extensiocongenital lung disease10010613AATDNL-OMON55372Kamada Ltd.70
尚未招募
3 期
Study To Know The Use of Trelagliptin Tablets in Diabetes PatientsCTRI/2022/10/046634Hetero Labs Limited
进行中（未招募）
不适用
A Phase III International Multi-center, Prospective, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Nitric Oxide Synthase Inhibition with Tilarginine Acetate Injection in Patients with Cardiogenic Shock Complicating Acute Myocardial Infarction,Or, the TRIUMPH Study:Tilarginine Acetate Injection in a Randomized International Study in Unstable AMI Patients / Cardiogenic Shock - TRIUMPH StudyEUCTR2005-000412-27-ATArginox Pharmaceuticals, Inc.700