Brief Title
A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency
Official Title
A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 144 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number of Participants With Adverse Events (AEs)
Condition
Alpha1-Antitrypsin Deficiency
Intervention
Alpha-1 15%
Study Arms / Comparison Groups
Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)
Description: Participants will receive Alpha-1 15% 72 mg/kg, single weekly subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
16
Start Date
August 13, 2021
Completion Date
February 5, 2023
Primary Completion Date
February 5, 2023
Eligibility Criteria
Inclusion Criteria: - Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (subjects with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor). - Have a documented pre-Alpha1-Proteinase Inhibitor (PI) augmentation therapy serum alpha-1 antitrypsin (AAT) level <11 micrometer (μM) (80 milligrams per decilitre (mg/dL) if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry). - Currently receiving Alpha1-PI augmentation therapy or has received Alpha1-PI augmentation therapy within the past. If the subject is currently receiving Alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of Alpha1-PI treatment, other than the IPs for this study, while participating in the study. Note: Subjects must not be naïve to Alpha1-PI augmentation therapy for study participation. - At the Screening Visit, have a post-bronchodilator forced expiratory volume (FEV1) ≥30% and <80% of predicted and FEV1/forced vital capacity (FVC) <70% (Global Initiative for Chronic Obstructive Lung Disease (GOLD) stage II or III). Exclusion Criteria: - Have had a moderate or severe Chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit. - Have history of lung or liver transplant. - Have any lung surgery during the past 2 years (excluding lung biopsy). - Have severe concomitant disease (example, congestive heart failure, clinically significant pulmonary fibrosis, malignant disease [except for skin cancers other than melanoma], history of acute hypersensitivity pneumonitis reaction, or current chronic hypersensitivity pneumonitis). - Females who are 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 (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence) throughout the study. - Have smoked during the past 6 months or a positive urine cotinine test at the Screening Visit that is due to smoking. - Participate in another Investigational product (IP) study within one month prior to the Week 1 (Baseline) Visit. - Have history of anaphylaxis or severe systemic response to any plasma-derived Alpha1-PI preparation or other blood product(s). - Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit. It is recommended to maintain the same dose throughout the study. - Use systemic or aerosolized antibiotics for a chronic COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit. - Have known selective or severe Immunoglobulin A (IgA) deficiency.
Gender
All
Ages
18 Years - 70 Years
Accepts Healthy Volunteers
No
Contacts
, +1 919 316 6693, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04722887
Organization ID
GC2008
Responsible Party
Sponsor
Study Sponsor
Grifols Therapeutics LLC
Study Sponsor
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Verification Date
January 2022