Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency

NCT02796937 | Enrolling By Invitation Phase 3

• 1 other identifier: GTi1201-OLE
• Study Type: interventional
• Target: 290
• Locations: 12 countries
• Sites: 28

Brief Summary

This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD).

Conditions

Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency

Keywords

Pulmonary Emphysema; Alpha-1 Antitrypsin Deficiency; AATD; Alpha-1 PI Deficiency; Alpha-1 Proteinase Inhibitor

Outcome Measures

Primary Outcomes (3)

• Adverse events (AEs)

  Monitoring of AEs

  Week 1 through Week 108

• Serious AEs (SAEs)

  Monitoring of SAEs

  Week 1 through Week 108

• Discontinuations from the study due to AEs

  Monitoring of discontinuations due to AEs

  Week 1 through Week 108

Secondary Outcomes (6)

• Change from baseline in whole lung PD15 (15th percentile point)

  Week 1 through Week 104

• Change from baseline in carbon monoxide diffusing capacity (DLco)

  Week 52 and Week 104

• Changes from baseline in forced expiratory volume in 1 second (FEV1)

  Week 52 and Week 104

• Change from baseline in Saint George's Respiratory Questionnaire

  Week 52 and Week 104

• Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations

  Week 2 through Week 108

Study Arms (1)

Alpha-1 MP

EXPERIMENTAL

Alpha-1 MP 60 mg/kg/week for up to 104 weeks

Biological: Alpha-1 MP

Interventions

Alpha-1 MP BIOLOGICAL

Alpha-1 MP 60 mg/kg/week for up to 104 weeks

Also known as: Prolastin-C

Alpha-1 MP

Eligibility Criteria

• Age: 20 Years - 72 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic).
• Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
• Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with \<1 year predicted overall survival.
• 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.
• 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.
• 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.
• 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.
• Has current evidence of chronic alcoholism or illicit drug abuse (addiction).
• Is currently participating in another investigational product (IP) study.
• Has a history of anaphylaxis or severe systemic response to any plasma- derived alpha1-PI preparation or other blood product(s).
• In the opinion of the investigator, is likely to have compliance problems with the protocol and the procedures of the protocol.
• 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.

Sponsors & Collaborators

• Grifols Therapeutics LLC: lead

Study Sites (28)

Grifols Investigative Site

Phoenix, Arizona, 85013, United States

Location

Grifols Investigative Site

Miami, Florida, 33136, United States

Location

Grifols Investigative Site

Wilmington, North Carolina, 28401, United States

Location

Grifols Investigative Site

Portland, Oregon, 97239, United States

Location

Grifols Investigative Site

Charleston, South Carolina, 29425, United States

Location

Grifols Investigative Site

Tyler, Texas, 75708, United States

Location

Grifols Investigative Site

Darlinghurst, New South Wales, 2010, Australia

Location

Grifols Investigative Site

Adelaide, 5000, Australia

Location

Grifols Investigative Site

Chermside, 4032, Australia

Location

Grifols Investigative Site

Fitzroy, 3065, Australia

Location

Grifols Investigative Site

Nedlands, 6009, Australia

Location

Grifols Investigative Site

Halifax, B3H 3A7, Canada

Location

Grifols Investigative Site

Toronto, M5T 3A9, Canada

Location

Grifols Investigative Site

Arhus C, 8000, Denmark

Location

Grifols Investigative Site

Hellerup, 2900, Denmark

Location

Grifols Investigative Site

Tallinn, 13419, Estonia

Location

Grifols Investigative Site

Turku, 20520, Finland

Location

Grifols Investigative Site

Bron, Rhone, 69677, France

Location

Grifols Investigative Site

Chisinau, 2025, Moldova

Location

Grifols Investigative Site

Auckland, 2025, New Zealand

Location

Grifols Investigative Site

Christchurch, 8011, New Zealand

Location

Grifols Investigative Site

Hamilton, 3200, New Zealand

Location

Grifols Investigative Site

Krakow, 31-066, Poland

Location

Grifols Investigative Site

Warsaw, 01-138, Poland

Location

Grifols Investigative Site

Barnaul, 656038, Russia

Location

Grifols Investigative Site

Gothenburg, 413 45, Sweden

Location

Grifols Investigative Site

Malmo, 20502, Sweden

Location

Grifols Investigative Site

Stockholm, 11361, Sweden

Location

MeSH Terms

Conditions

Pulmonary Emphysema; alpha 1-Antitrypsin Deficiency

Interventions

Mp alpha1 receptor

Condition Hierarchy (Ancestors)

Pulmonary Disease, Chronic Obstructive; Lung Diseases, Obstructive; Lung Diseases; Respiratory Tract Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Liver Diseases; Digestive System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2016
• First Posted: June 13, 2016
• Study Start: July 1, 2016
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): February 1, 2029
• Last Updated: March 4, 2026

Record last verified: 2026-03

Locations

AU (5); FI (1); SE (3); DK (2); ES (1); NZ (3); PL (2); RU (1); CA (2); MO (1); US (6); FR (1)