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The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alpha-1 Proteinase Inhibitor (Human), modified process | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| alpha-1 proteinase inhibitor (human) | Drug | 60 mg/kg weekly for 20 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kim Hanna, MSc | Grifols Therapeutics LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Jewish Medical and Research Center | Denver | Colorado | 80206 | United States | ||
| University of Florida College of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 3494198 | Background | 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. | |
| 7028785 | Background | 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. |
| Label | URL |
|---|---|
| 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 | View source |
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First-subject-first-dose was 12 June 2006, last-subject-last-visit was 20 March 2007. The study was performed in 10 clinical sites, 8 sites in the United States and 2 sites in the United Kingdom.
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| ID | Title | Description |
|---|---|---|
| FG000 | Alpha-1MP Treatment Group | All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 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 |
| 6169740 | Background | 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. |
| 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 | View source |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Entered Study |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 38 subjects received study medication and one subject discontinued from the study due to an AE. Therefore, 37 subjects completed the study. 18 subjects were naive ((i.e., never having received previous Alpha-1 protease inhibitor augmentation therapy) and 19 subjects were non-naive. | Posted | Sep 2009 | Number | Participants | 24 weeks |
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Alpha-1MP Treatment Group | All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks | 2 | 38 | 26 | 38 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations |
| |||
| Rash | Skin and subcutaneous tissue disorders |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders |
| |||
| Chest pain | General disorders |
| |||
| Influenza like illness | General disorders |
| |||
| Chills | General disorders |
| |||
| Nasopharyngitis | Infections and infestations |
| |||
| Oral candidiasis | Infections and infestations |
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| Upper respiratory tract infection | Infections and infestations |
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| Urinary tract infection | Infections and infestations |
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| Arthralgia | Musculoskeletal and connective tissue disorders |
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| Back pain | Musculoskeletal and connective tissue disorders |
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| Muscle spasms | Musculoskeletal and connective tissue disorders |
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| Dizziness | Nervous system disorders |
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| Headache | Nervous system disorders |
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| Anxiety | Psychiatric disorders |
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| Cough | Respiratory, thoracic and mediastinal disorders |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders |
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| Pharyngolaryngeal pain | Respiratory, thoracic and mediastinal disorders |
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| Hot flush | Vascular disorders |
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The investigator must send a draft manuscript of the publication or abstract to the sponsor, thirty days in advance of submission, in order to obtain approval prior to submission of the final version for publication. This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution that satisfies both parties.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Henry Li | Grifols Therapeutics | 1-800-520-2807 | henry.li@grifols.com |
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D011656 | Pulmonary Emphysema |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D029424 | Pulmonary Disease, Chronic Obstructive |
| D008173 | Lung Diseases, Obstructive |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
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| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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| Title | Measurements |
|---|---|
|
| Alpha-1 MP subjects with ≥1 drug-related TEAE |
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