The Safety and Tolerability of Alpha-1 MP In Subjects With Alpha-1-Antitrypsin (AAT) Deficiency - Full Text View

Sponsor:	Talecris Biotherapeutics
Information provided by:	Talecris Biotherapeutics
ClinicalTrials.gov Identifier:	NCT00301366

Purpose

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

Condition	Intervention	Phase
Alpha 1-Antitrypsin Deficiency	Drug: alpha-1 proteinase inhibitor (human)	Phase III

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Multi-Center, Open-Label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-Antitrypsin (AAT) Deficiency

Resource links provided by NLM:

Genetics Home Reference related topics: alpha-1 antitrypsin deficiency

MedlinePlus related topics: Alpha-1 Antitrypsin Deficiency Emphysema

U.S. FDA Resources

Further study details as provided by Talecris Biotherapeutics:

Primary Outcome Measures:

treatment-emergent adverse events [ Time Frame: 24 weeks ]

Secondary Outcome Measures:

all adverse events [ Time Frame: 24 weeks ]
Laboratory data outside normal range [ Time Frame: 24 weeks ]

Enrollment:	38
Study Start Date:	May 2006
Study Completion Date:	March 2007

Arms	Assigned Interventions
Experimental: Entered study	Drug: alpha-1 proteinase inhibitor (human) 60 mg/kg weekly for 20 weeks Other Names: Prolastin Alpha-1 antitrypsin (AAT) BAY x 5747 BAY 10-5233 TAL-05-00007

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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Documented diagnosis of congenital Alpha1-antitrypsin deficiency
Documented 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00301366

Locations

United States, Colorado
National Jewish Medical and Research Center
Denver, Colorado, United States, 80206
United States, Florida
University of Florida College of Medicine
Gainesville, Florida, United States, 32610-0225
University of Miami School of Medicine
Miami, Florida, United States, 33101
United States, New York
St Lukes-Roosevelt Hospital Center, New York
New York, New York, United States, 10019
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44122
United States, Pennsylvania
Temple University Hospital
Philadelphia, Pennsylvania, United States, 19140
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Texas
University of Texas Health Center at Tyler
Tyler, Texas, United States, 75708-3154
Poland
Uniwersytetu Medycznego w Lodzi, Klinika Pneumonologii i Alergologii
Lodz, Poland, 90 - 153
United Kingdom
University of Cambridge - Cambridge Institute for Medical Research
Cambridge, England, United Kingdom, CB2 2XY
University Teaching Hospital of Edinburgh
Edinburgh, Scotland, United Kingdom, EH8 9AG

Sponsors and Collaborators

Talecris Biotherapeutics

Investigators

Study Director:

Eric Batson, MD

Talecris Biotherapeutics

More Information

Additional Information:

The "Alpha-1 Foundation", dedicated to providing the leadership and resources that will result in increased research, improved health, worldwide detection and a cure for Alpha-1 Antitrypsin Deficiency This link exits the ClinicalTrials.gov site

This link exits the ClinicalTrials.gov site

AlphaNet, Inc devoted to improving the lives of individuals with Alpha-1 antitrypsin deficiency through comprehensive disease management services, clinical research administration, and consultative services This link exits the ClinicalTrials.gov site

Publications:

Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987 Apr 23;316(17):1055-62.

Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981 Nov;68(5):1158-65.

Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J Clin Invest. 1981 Oct;68(4):889-98.

ClinicalTrials.gov Identifier:	NCT00301366 History of Changes
Other Study ID Numbers:	11815
Study First Received:	March 8, 2006
Last Updated:	September 26, 2008
Health Authority:	United States: Food and Drug Administration; Netherlands: Medicines Evaluation Board (MEB); Poland: Ministry of Health; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Talecris Biotherapeutics:

alpha 1-Antitrypsin Deficiency
alpha 1-Antitrypsin
pulmonary emphysema

Additional relevant MeSH terms:

Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes

Alpha 1-Antitrypsin
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012