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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00301366
Recruitment Status : Completed
First Posted : March 10, 2006
Results First Posted : August 19, 2014
Last Update Posted : August 19, 2014
Information provided by (Responsible Party):
Grifols Therapeutics LLC

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.

Condition or disease Intervention/treatment Phase
Alpha 1-Antitrypsin Deficiency Drug: alpha-1 proteinase inhibitor (human) Phase 3

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : June 2006
Actual Primary Completion Date : March 2007
Actual Study Completion Date : March 2007

Arm Intervention/treatment
Experimental: Alpha-1 Proteinase Inhibitor (Human), modified process
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.
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

Primary Outcome Measures :
  1. Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. [ Time Frame: 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00301366

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
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
Grifols Therapeutics LLC
Study Director: Kim Hanna, MSc Grifols Therapeutics LLC

Additional Information:
Responsible Party: Grifols Therapeutics LLC Identifier: NCT00301366     History of Changes
Other Study ID Numbers: 11815
First Posted: March 10, 2006    Key Record Dates
Results First Posted: August 19, 2014
Last Update Posted: August 19, 2014
Last Verified: July 2014

Keywords provided by Grifols Therapeutics LLC:
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
Pathologic Processes
Alpha 1-Antitrypsin
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action