Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0 Level: LLT Classification code 10014563 Term: Emphysema pulmonary System Organ Class: 100000004855
Conditions
Interventions
Trade Name: Prolastin-C
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
CAS Number: 9041-92-3
Current Sponsor code: GTI1201-O
Sponsors
Grifols Therapeutics LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 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. 2. Is willing and able to provide informed consent. 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) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: 1. Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic). 2. Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis. 3. Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with <1 year predicted overall survival. 4. 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. 5. 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. 6. 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. 7. 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. 8. Has current evidence of chronic alcoholism or illicit drug abuse (addiction). 9. Is currently participating in another investigational product (IP) study. 10. Has a history of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s). 11. In the opinion of the investigator, is likely to have compliance problems with the protocol and the procedures of the protocol. 12. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha-1 MP 60 mg/kg/week in subjects with AATD.;Secondary Objective: The secondary objective of this study is to obtain an additional 2 years of data on disease progression in subjects with AATD intravenously administered Alpha-1 MP 60 mg/kg/week.;Primary end point(s): Adverse events and lab parameters;Timepoint(s) of evaluation of this end point: Every study visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Lung density from CT densitometry;Timepoint(s) of evaluation of this end point: Weeks 52, 104 and Early Discontinuation | — |
Countries
Argentina, Australia, Brazil, Canada, Denmark, Estonia, Finland, France, Germany, New Zealand, Poland, Romania, Russian Federation, Spain, Sweden, United States
Contacts
Public ContactSusan Sorrells, Clinical Director
Grifols Therapeutics LLC
Outcome results
None listed