The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Phase 3

Completed

Brief Summary: The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.

Detailed Description: The objective of this clinical trial (STAMP: Safety and Tolerability of Alpha-1 Modified Process) is to study the safety and tolerability of Alpha-1 MP in adult Alpha 1-antitrypsin deficient subjects as reported over 20 weeks of therapy. The primary objective is to describe the nature and frequency of treatment-emergent adverse events with "treatment-emergent" defined as any adverse event occurring after the start of the first study drug infusion.

Recruitment & Eligibility

Inclusion Criteria

Documented diagnosis of congenital Alpha1-antitrypsin deficiency
Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted value within last 6 months.
Signed written informed consent prior to initiation of any study related procedures.

Exclusion Criteria

Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study
Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids used on a routine or as needed basis).
Subjects who have had exacerbations of their disease within one month of trial entry

Arm && Interventions

Group	Intervention	Description
Alpha-1 Proteinase Inhibitor (Human), modified process	alpha-1 proteinase inhibitor (human)	Study the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects.

Primary Outcome Measures

Name	Time	Method
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.	24 weeks	An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.

Secondary Outcome Measures

Name	Time	Method

Locations (10): University of Florida College of Medicine
🇺🇸
Gainesville, Florida, United States
Cleveland Clinic Foundation
🇺🇸
Cleveland, Ohio, United States
University of Cambridge - Cambridge Institute for Medical Research
🇬🇧
Cambridge, England, United Kingdom
University Teaching Hospital of Edinburgh
🇬🇧
Edinburgh, Scotland, United Kingdom
Medical University of South Carolina
🇺🇸
Charleston, South Carolina, United States
St Lukes-Roosevelt Hospital Center, New York
🇺🇸
New York, New York, United States
National Jewish Medical and Research Center
🇺🇸
Denver, Colorado, United States
University of Miami School of Medicine
🇺🇸
Miami, Florida, United States
Temple University Hospital
🇺🇸
Philadelphia, Pennsylvania, United States
University of Texas Health Center at Tyler
🇺🇸
Tyler, Texas, United States