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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 : August 27, 2019
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Tracking Information
First Submitted Date  ICMJE June 3, 2016
First Posted Date  ICMJE June 13, 2016
Last Update Posted Date August 27, 2019
Study Start Date  ICMJE July 2016
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 7, 2018)
  • Adverse events (AEs) [ Time Frame: Week 1 through Week 108 ]
    Monitoring of AEs
  • Serious AEs (SAEs) [ Time Frame: Week 1 through Week 108 ]
    Monitoring of SAEs
  • Discontinuations from the study due to AEs [ Time Frame: Week 1 through Week 108 ]
    Monitoring of discontinuations due to AEs
Original Primary Outcome Measures  ICMJE
 (submitted: June 7, 2016)
  • Incidence of adverse events (AEs) [ Time Frame: Week 1 through Week 108 ]
  • Incidence of serious adverse events (SAEs) [ Time Frame: Week 1 through Week 108 ]
  • Number of subjects with changes from baseline in vital signs parameters [ Time Frame: Week 26 through Week 108 ]
    To include changes from low, normal, or high at baseline to low, normal, or high post-baseline
  • Number of subjects with changes from baseline in hematology parameters [ Time Frame: Week 26 through Week 108 ]
    To include changes from low, normal, or high at baseline to low, normal, or high post-baseline
  • Number of subjects with changes from baseline in chemistry parameters [ Time Frame: Week 26 through Week 108 ]
    To include changes from low, normal, or high at baseline to low, normal, or high post-baseline
Change History Complete list of historical versions of study NCT02796937 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2018)
  • 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
  • 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
  • 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
  • Change from baseline in Saint George's Respiratory Questionnaire [ Time Frame: Week 52 and Week 104 ]
    Health-related quality of life assessment tool
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: June 7, 2016)
  • Change from baseline in total lung capacity-adjusted lung density [ Time Frame: Week 1 through Week 104 ]
  • Change from baseline in carbon monoxide diffusing capacity [ Time Frame: Week 52 and Week 104 ]
  • Changes from baseline in forced expiratory volume in 1 second [ Time Frame: Week 52 and Week 104 ]
  • Change from baseline in Saint George's Respiratory Questionnaire [ Time Frame: Week 52 and Week 104 ]
  • Incidence and severity of Chronic Obstructive Pulmonary Disease exacerbations [ Time Frame: Week 2 through Week 108 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
Official Title  ICMJE 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
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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
Intervention  ICMJE Biological: Alpha-1 MP
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
Other Name: Prolastin-C
Study Arms  ICMJE Experimental: Alpha-1 MP
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
Intervention: Biological: Alpha-1 MP
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: June 7, 2016)
250
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2025
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 20 Years to 72 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Denmark,   Estonia,   New Zealand,   Poland,   Sweden,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02796937
Other Study ID Numbers  ICMJE GTi1201-OLE
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Grifols Therapeutics LLC
Study Sponsor  ICMJE Grifols Therapeutics LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Grifols Therapeutics LLC
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP