Long-term safety of Prolastin®-C, an alpha1-proteinase inhibitor, in Japanese patients with alpha1-antitrypsin deficiency.

Background: Safety and pharmacokinetics (PK) of alpha-proteinase inhibitor, modified process (Alpha-1 MP), was evaluated in a clinical trial of Japanese patients with alpha-antitrypsin deficiency (AATD). The present study aimed to evaluate the long-term safety of weekly intravenous infusions of 60 mg/kg Alpha-1 MP in Japanese patients with AATD.

Methods: This was a multi-center, open-label extension (OLE) study that enrolled adult patients with AATD, who had completed the preceding safety and PK clinical trial.

Safety and pharmacokinetics of Alpha-1 MP (Prolastin-C) in Japanese patients with alpha-antitrypsin (AAT) deficiency.

Background: Alpha-Proteinase Inhibitor, Modified Process (Alpha-1 MP) is used for augmentation therapy in alpha1-antitrypsin deficiency (AATD), an extremely rare disease in Japan. Weekly doses of 60 mg/kg Alpha-1 MP have been shown to be safe and well tolerated in non-Japanese subjects, but the safety and pharmacokinetics (PK) have not been evaluated in Japanese subjects. The objectives of this study were to evaluate the safety and PK of 60 mg/kg Alpha-1 MP administered by weekly IV infusions over 8 weeks in Japanese subjects with AATD.

Bioequivalence of a Liquid Formulation of Alpha-Proteinase Inhibitor Compared with Prolastin®-C (Lyophilized Alpha-PI) in Alpha-Antitrypsin Deficiency.

This study evaluated the bioequivalence, safety, and immunogenicity of a new liquid formulation of human plasma-derived alpha-proteinase inhibitor, Liquid Alpha-PI, compared with the Lyophilized Alpha-PI formulation (Prolastin®-C), for augmentation therapy in patients with alpha-antitrypsin deficiency (AATD). In this double-blind, randomized, 20-week crossover study, 32 subjects with AATD were randomized to receive 8 weekly infusions of 60 mg/kg of Liquid Alpha-PI or Lyophilized Alpha-PI. Serial blood samples were drawn for 7 days after the last dose followed by 8 weeks of the alternative treatment.

SPARTA clinical trial design: exploring the efficacy and safety of two dose regimens of alpha1-proteinase inhibitor augmentation therapy in alpha1-antitrypsin deficiency.

Background: Alpha1-antitrypsin deficiency (AATD) is an underdiagnosed genetic disorder that results in early-onset emphysema due to low serum levels of alpha1-proteinase inhibitor (alpha1-PI), leading to increased activity of tissue-damaging neutrophil elastase. Clinical outcomes of AATD may be improved by administering alpha1-PI augmentation therapy. Here, we describe the design of the ongoing Study of ProlAstin-c Randomized Therapy with Alpha-1 augmentation (SPARTA), a phase 3 trial designed to evaluate progression of lung tissue loss in patients with severe AATD receiving human alpha1-PI (Prolastin(®)-C) versus placebo, using whole-lung computed tomography (CT) densitometry.

Brief report: Assessment of children's gastrointestinal symptoms for clinical trials.

Objective: To conduct a pilot study evaluating a procedure for assessment of daily symptoms and functioning in pediatric patients.

Method: Participants included 11 parent-child dyads referred to a tertiary care center for evaluation of constipation and abdominal pain. Each family was provided a hand-held computer and modem.