alpha-1-proteinase inhibitor (human) for Pulmonary emphysema due to alpha-1 antitrypsin deficiency

Inclusion criteria

• {"criterion_text":"- a. Has completed participation in Study GTi1201 (ie, completed Week 156 and Week 160/End-of-Study Visit) Or b. Has experienced a decline in FEV1 at the annualized rate of ≥134.4 mL/year at or after the Week 104 Visit in GTi1201."}
• {"criterion_text":"- Is willing and able to provide informed consent."}

Exclusion criteria

• {"criterion_text":"- Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic)."}
• {"criterion_text":"- Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis."}
• {"criterion_text":"- Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with <1 year predicted overall survival"}
• {"criterion_text":"- Has a metal object (newly received since starting GTi1201) that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis."}
• {"criterion_text":"- Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception; placement of an intrauterine device or intrauterine system; condom or occlusive cap with spermicidal foam/gel/film/cream/suppository; male sterilization; or abstinence) throughout the study."}
• {"criterion_text":"- Has clinical signs and symptoms of viral infection requiring virus safety testing at the Week 160/End-of-Study Visit or Early Discontinuation Visit in Study GTi1201, and the virus safety test results are indicative of acute or chronic infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV), or parvovirus B19 (B19V). Note: If the virus safety test results are indicative of acute or chronic infection with HAV, HBV, HCV, HIV, or B19V, the subject will be considered a screen failure and must be withdrawn from the study."}
• {"criterion_text":"- Has current evidence of smoking, which includes electronic/vapor cigarettes, or has a positive urine cotinine test at the Week 160/End-of-Study Visit in Study GTi1201 that is due to smoking."}
• {"criterion_text":"- Has current evidence of chronic alcoholism or illicit drug abuse (addiction)."}
• {"criterion_text":"- Is currently participating in another investigational product (IP) study."}
• {"criterion_text":"- Has a history of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s)."}
• {"criterion_text":"- In the opinion of the investigator, is likely to have compliance problems with the protocol and the procedures of the protocol."}
• {"criterion_text":"- Has any medical condition that the investigator feels might confound the results of the study or pose an additional risk to the subject during study participation."}

Primary endpoints

• {"endpoint_text":"- Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry\n- Carbon monoxide diffusing capacity (DLCO)\n- forced expiratory volume in 1 second (FEV1)\n- St. George’s Respiratory Questionnaire (SGRQ) score\n- Five level EQ-5D (EQ-5D-5L) score\n- Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria","definition_or_measurement_approach":"PD15 measured using computed tomography (CT) densitometry; DLCO measured as carbon monoxide diffusing capacity; FEV1 measured as forced expiratory volume in 1 second; SGRQ score and EQ-5D-5L score assessed by the respective validated questionnaires; COPD exacerbations assessed by incidence and severity defined by ATS/ERS criteria."}

Secondary endpoints

• {"endpoint_text":"- AEs, serious AEs (SAEs), and discontinuations due to AEs and SAEs\n- Physical examination (complete and respiratory)\n- Clinical laboratory parameters: hematology, chemistry (includes high-sensitivity C-reactive protein [hs-CRP])","definition_or_measurement_approach":"Adverse events (AEs) and serious AEs recorded per standard safety reporting; physical examinations include complete and respiratory assessments; clinical laboratory parameters include hematology and chemistry panels with hs-CRP."}

Sweden

Latest Decision Or Authorization Date

21-08-2024

Number Of Sites

2

Number Of Participants

30

Denmark

Latest Decision Or Authorization Date

22-08-2024

Number Of Sites

1

Number Of Participants

42

Poland

Latest Decision Or Authorization Date

16-09-2024

Number Of Sites

2

Number Of Participants

43

Estonia

Latest Decision Or Authorization Date

23-08-2024

Number Of Sites

1

Number Of Participants

3

France

Latest Decision Or Authorization Date

27-08-2024

Number Of Sites

1

Number Of Participants

1

Finland

Latest Decision Or Authorization Date

21-08-2024

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Grifols Therapeutics LLC

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

IQVIA Limited

Responsibilities

Various sponsor duties (codes: 1,12,2,6,7); contact email eu_clinical_trials_information@iqvia.com; phone 441184506016

Third parties

• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: 1,12,2,6,7; contact email: eu_clinical_trials_information@iqvia.com; phone: 441184506016","organisation_type":"Pharmaceutical company"}