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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2024
• Main ID: NCT02796937
• Date of registration: 03/06/2016
• Prospective Registration: Yes
• Primary sponsor: Grifols Therapeutics LLC
• Public title: Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency SPARTA-OLE
• Scientific title: An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
• Date of first enrolment: July 2016
• Target sample size: 290
• Recruitment status: Enrolling by invitation
• URL: https://clinicaltrials.gov/ct2/show/NCT02796937
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Denmark, Estonia, Finland, France, New Zealand, Poland, Russian Federation, Sweden, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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.



Age minimum: 20 Years
Age maximum: 72 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency

Intervention(s)

Biological: Alpha-1 MP

Primary Outcome(s)

Serious AEs (SAEs) [Time Frame: Week 1 through Week 108]

Adverse events (AEs) [Time Frame: Week 1 through Week 108]

Discontinuations from the study due to AEs [Time Frame: Week 1 through Week 108]

Secondary Outcome(s)

Changes from baseline in forced expiratory volume in 1 second (FEV1) [Time Frame: Week 52 and Week 104]

Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations [Time Frame: Week 2 through Week 108]

Change from baseline in Saint George's Respiratory Questionnaire [Time Frame: Week 52 and Week 104]

Change from baseline in the EQ-5D-5L Questionnaire [Time Frame: Week 52 and Week 104]

Change from baseline in whole lung PD15 (15th percentile point) [Time Frame: Week 1 through Week 104]

Change from baseline in carbon monoxide diffusing capacity (DLco) [Time Frame: Week 52 and Week 104]

Secondary ID(s)

GTi1201-OLE

Source(s) of Monetary Support

Please refer to primary and secondary sponsors