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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Conditions
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Alpha 1-Antitrypsin Deficiency

Intervention / Treatment
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* Drug: alpha-1 proteinase inhibitor (human)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Alpha-1 Proteinase Inhibitor (Human), modified process<br>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)<br>* 60 mg/kg weekly for 20 weeks<br>* Other names: TAL-05-00007;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema