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

Primary Purpose

Alpha 1-Antitrypsin Deficiency

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
alpha-1 proteinase inhibitor (human)
Sponsored by
Grifols Therapeutics LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha 1-Antitrypsin Deficiency focused on measuring alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • National Jewish Medical and Research Center
  • University of Florida College of Medicine
  • University of Miami School of Medicine
  • St Lukes-Roosevelt Hospital Center, New York
  • Cleveland Clinic Foundation
  • Temple University Hospital
  • Medical University of South Carolina
  • University of Texas Health Center at Tyler
  • University of Cambridge - Cambridge Institute for Medical Research
  • University Teaching Hospital of Edinburgh

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Alpha-1 Proteinase Inhibitor (Human), modified process

Arm Description

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.

Outcomes

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.
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

Full Information

First Posted
March 8, 2006
Last Updated
July 30, 2014
Sponsor
Grifols Therapeutics LLC
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1. Study Identification

Unique Protocol Identification Number
NCT00301366
Brief Title
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
Acronym
STAMP
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 Type
Interventional

2. Study Status

Record Verification Date
July 2014
Overall Recruitment Status
Completed
Study Start Date
June 2006 (undefined)
Primary Completion Date
March 2007 (Actual)
Study Completion Date
March 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Grifols Therapeutics LLC

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha 1-Antitrypsin Deficiency
Keywords
alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
38 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Alpha-1 Proteinase Inhibitor (Human), modified process
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
alpha-1 proteinase inhibitor (human)
Other Intervention Name(s)
Prolastin, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233, TAL-05-00007
Intervention Description
60 mg/kg weekly for 20 weeks
Primary Outcome Measure Information:
Title
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.
Description
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.
Time Frame
24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kim Hanna, MSc
Organizational Affiliation
Grifols Therapeutics LLC
Official's Role
Study Director
Facility Information:
Facility Name
National Jewish Medical and Research Center
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
University of Florida College of Medicine
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610-0225
Country
United States
Facility Name
University of Miami School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33101
Country
United States
Facility Name
St Lukes-Roosevelt Hospital Center, New York
City
New York
State/Province
New York
ZIP/Postal Code
10019
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44122
Country
United States
Facility Name
Temple University Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19140
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
University of Texas Health Center at Tyler
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708-3154
Country
United States
Facility Name
University of Cambridge - Cambridge Institute for Medical Research
City
Cambridge
State/Province
England
ZIP/Postal Code
CB2 2XY
Country
United Kingdom
Facility Name
University Teaching Hospital of Edinburgh
City
Edinburgh
State/Province
Scotland
ZIP/Postal Code
EH8 9AG
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
3494198
Citation
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. doi: 10.1056/NEJM198704233161704.
Results Reference
background
PubMed Identifier
7028785
Citation
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. doi: 10.1172/jci110360.
Results Reference
background
PubMed Identifier
6169740
Citation
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. doi: 10.1172/jci110344.
Results Reference
background
Links:
URL
http://www.alphaone.org
Description
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
URL
http://www.alphanet.org
Description
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

Learn more about this trial

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

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