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Clinical Trials/2024-513962-20-00
2024-513962-20-00
Active, not recruiting
Phase 3

An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alphal-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency

Grifols Therapeutics LLC8 sites in 6 countries120 target enrollmentStarted: August 21, 2024Last updated:
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Overview

Phase
Phase 3
Status
Active, not recruiting
Enrollment
120
Locations
8
Primary Endpoint
The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria
OverviewEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

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.

Eligibility Criteria

Ages
18 years to 65+ years (18-64 Years, 65+ Years)
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • •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 GTi
  • •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 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.
  • •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.

Outcomes

Primary Outcomes

The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria

The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria

Secondary Outcomes

  • The following safety variables will be assessed in this study: •AEs, serious AEs (SAEs), and discontinuations due to AEs and SAEs •Physical examination (complete and respiratory) •Clinical laboratory parameters: hematology, chemistry (includes high-sensitivity C-reactive protein [hs-CRP])

Investigators

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

SCIENTIFIC AND INNOVATION OFFICE DRUG DEVELOPMENT

Scientific

Grifols Therapeutics LLC

Study Sites (8)

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