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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT00301366
First received: March 8, 2006
Last updated: July 30, 2014
Last verified: July 2014
  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 3

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:


Further study details as provided by Grifols Therapeutics Inc.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    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.


Enrollment: 38
Study Start Date: June 2006
Study Completion Date: March 2007
Primary Completion Date: March 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
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 Inc.
Investigators
Study Director: Kim Hanna, MSc Grifols Therapeutics Inc.
  More Information

Additional Information:
Publications:
Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT00301366     History of Changes
Other Study ID Numbers: 11815
Study First Received: March 8, 2006
Results First Received: August 28, 2009
Last Updated: July 30, 2014
Health Authority: United States: Food and Drug Administration
Netherlands: Medicines Evaluation Board (MEB)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Grifols Therapeutics Inc.:
alpha 1-Antitrypsin Deficiency
alpha 1-Antitrypsin
pulmonary emphysema

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Digestive System Diseases
Emphysema
Genetic Diseases, Inborn
Liver Diseases
Lung Diseases
Pathologic Processes
Respiratory Tract Diseases
Subcutaneous Emphysema
Alpha 1-Antitrypsin
Protease Inhibitors
Protein C Inhibitor
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Serine Proteinase Inhibitors
Trypsin Inhibitors

ClinicalTrials.gov processed this record on November 20, 2014