The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
STAMP
Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency
1 other identifier
interventional
38
2 countries
10
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jun 2006
Shorter than P25 for phase_3
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 8, 2006
CompletedFirst Posted
Study publicly available on registry
March 10, 2006
CompletedStudy Start
First participant enrolled
June 1, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2007
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2007
CompletedResults Posted
Study results publicly available
August 19, 2014
CompletedAugust 19, 2014
July 1, 2014
9 months
March 8, 2006
August 28, 2009
July 30, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
24 weeks
Study Arms (1)
Alpha-1 Proteinase Inhibitor (Human), modified process
EXPERIMENTALStudy 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.
Interventions
60 mg/kg weekly for 20 weeks
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
National Jewish Medical and Research Center
Denver, Colorado, 80206, United States
University of Florida College of Medicine
Gainesville, Florida, 32610-0225, United States
University of Miami School of Medicine
Miami, Florida, 33101, United States
St Lukes-Roosevelt Hospital Center, New York
New York, New York, 10019, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44122, United States
Temple University Hospital
Philadelphia, Pennsylvania, 19140, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
University of Texas Health Center at Tyler
Tyler, Texas, 75708-3154, United States
University of Cambridge - Cambridge Institute for Medical Research
Cambridge, England, CB2 2XY, United Kingdom
University Teaching Hospital of Edinburgh
Edinburgh, Scotland, EH8 9AG, United Kingdom
Related Publications (3)
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.
PMID: 3494198BACKGROUNDGadek 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.
PMID: 7028785BACKGROUNDGadek 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.
PMID: 6169740BACKGROUND
Related Links
- 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
- 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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Henry Li
- Organization
- Grifols Therapeutics
Study Officials
- STUDY DIRECTOR
Kim Hanna, MSc
Grifols Therapeutics LLC
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 8, 2006
First Posted
March 10, 2006
Study Start
June 1, 2006
Primary Completion
March 1, 2007
Study Completion
March 1, 2007
Last Updated
August 19, 2014
Results First Posted
August 19, 2014
Record last verified: 2014-07