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Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency (SPARTA-OLE)

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ClinicalTrials.gov Identifier: NCT02796937
Recruitment Status : Enrolling by invitation
First Posted : June 13, 2016
Last Update Posted : March 13, 2023
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Study Description
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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).

Condition or disease Intervention/treatment Phase
Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency Biological: Alpha-1 MP Phase 3

Detailed Description:

This is a 2-year open-label 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 Alpha-1 MP 60 mg/kg/week in subjects with AATD.

The study consists of a Screening Visit (the same visit as the End-of-Study Visit in the GTi1201 study for subjects who complete the GTi1201 study or is the same visit as the Early Discontinuation Visit for subjects meeting the early withdrawal criterion for forced expiratory volume in 1 second [FEV1] decline), a treatment period of 104 weeks (beginning immediately after screening [on the same day as the Screening Visit] but no sooner than 1 week after the last infusion of investigational product in the GTi1201 study), and an End-of-Study Visit.

Subjects meeting the entrance criteria of the extension study will begin receiving weekly intravenous (IV) infusions of Alpha-1 MP 60 mg/kg on the day of screening and will continue to receive weekly infusions for a total of 104 infusions.

Safety assessments will include adverse events, concomitant medications, complete physical examination (excluding breast and genitourinary examination), hematology, chemistry, urine cotinine, and pregnancy test. Efficacy assessments will include whole lung computed tomography density, quality-of-life assessment, carbon monoxide diffusing capacity, and pulmonary function tests. The occurrence of chronic obstructive pulmonary exacerbations, will also be evaluated as a safety and as an efficacy measurement.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : July 2016
Estimated Primary Completion Date : April 2028
Estimated Study Completion Date : April 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Alpha-1 MP
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
 Biological: Alpha-1 MP
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
Other Name: Prolastin-C


Outcome Measures
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Primary Outcome Measures :
  1. Adverse events (AEs) [ Time Frame: Week 1 through Week 108 ]
    Monitoring of AEs

  2. Serious AEs (SAEs) [ Time Frame: Week 1 through Week 108 ]
    Monitoring of SAEs

  3. Discontinuations from the study due to AEs [ Time Frame: Week 1 through Week 108 ]
    Monitoring of discontinuations due to AEs


Secondary Outcome Measures :
  1. Change from baseline in whole lung PD15 (15th percentile point) [ Time Frame: Week 1 through Week 104 ]
    Whole lung PD15 measured by computed tomography scans

  2. Change from baseline in carbon monoxide diffusing capacity (DLco) [ Time Frame: Week 52 and Week 104 ]
    DLco performed according to American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines

  3. Changes from baseline in forced expiratory volume in 1 second (FEV1) [ Time Frame: Week 52 and Week 104 ]
    FEV1 performed according to ATS/ERS guidelines

  4. Change from baseline in Saint George's Respiratory Questionnaire [ Time Frame: Week 52 and Week 104 ]
    Health-related quality of life assessment tool

  5. Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations [ Time Frame: Week 2 through Week 108 ]
    Severe COPD exacerbations as defined by ATS/ERS guidelines

  6. Change from baseline in the EQ-5D-5L Questionnaire [ Time Frame: Week 52 and Week 104 ]
    Heath-related quality of life assessment tool


Eligibility Criteria
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Ages Eligible for Study:   20 Years to 72 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02796937


Locations
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Sponsors and Collaborators
Grifols Therapeutics LLC
More Information
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Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT02796937    
Other Study ID Numbers: GTi1201-OLE
First Posted: June 13, 2016    Key Record Dates
Last Update Posted: March 13, 2023
Last Verified: March 2023
Keywords provided by Grifols Therapeutics LLC:
Pulmonary Emphysema
Alpha-1 Antitrypsin Deficiency
AATD
Alpha-1 PI Deficiency
Alpha-1 Proteinase Inhibitor
Additional relevant MeSH terms:
Layout table for MeSH terms
Alpha 1-Antitrypsin Deficiency
Pulmonary Emphysema
Emphysema
Pathologic Processes
Pulmonary Disease, Chronic Obstructive
Lung Diseases, Obstructive
Lung Diseases
Respiratory Tract Diseases
Liver Diseases
 Digestive System Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Alpha 1-Antitrypsin
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action