Alpha-1 Antitrypsin (AAT) deficiency MedDRA version: 9 Level: LLT Classification code 10001806
Conditions
Interventions
Product Name: Alpha1-proteinase inhibitor (Human), modified process (Alpha-1 MP)
Product Code: TAL-05-00007
Pharmaceutical Form: Powder for solution for infusion
Sponsors
Talecris Biotherapeutics
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented diagnosis of congenital Alpha1-antitrypsin deficiency with genotype being PiZZ, PiZ(null), Pi(null)(null), or “At-risk” alleles as listed in Appendices 10.3. of the Study Protocol. The subject’s genotype must be documented by and/or verifiable through the Alpha-1 Antitrypsin Genetics Laboratory database located in Gainesville, Florida. If a subject's genotype is not documented and or verifiable by the Alpha-1 Antitrypsin Genetics Laboratory, the subject will be required, at screening to provide an additional blood sample (two 10-mL EDTA tubes)for analysis by the Alpha-1 Antitrypsin Genetics Laboratory. This analysis will include genotyping, and if the subject is treatment-naïve, a baseline serum Alpha-1 Proteinase Inhibitor level. 2. Documented Alpha-1 Proteinase Inhibitor serum levels =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The following exclusion criteria must be evaluated at screening: 1. Diagnosis of liver cirrhosis; 2. Severe concomitant disease (e.g. congestive heart failure (NYHA III/IV), clinically significant pulmonary fibrosis, serious malignant disease); 3. Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study; 4. Within the last month prior to study entry, participation in another clinical study if the subject received any investigational product during his/her participation in the investigational study. 5. History of anaphylaxis or severe systemic response to plasma-derived Alpha1-Proteinase Inhibitor or other blood product(s); 6. Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids). Self-limited therapy with systemic steroids to treat an adverse event is not cause to discontinue or disrupt Alpha-1 PI therapy unless, in the opinion of the investigator, this action is warranted. The study’s Medical Monitor should be consulted if the subject requires frequent (>2) self-limited courses of systemic steroids or if long-term therapy is warranted during the subject’s participation in the study. 7. Known selective IgA deficiency 8. Mentally challenged adult subjects who cannot give independent informed consent; 9. Subjects who have had exacerbations of their disease within one month of trial entry;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this clinical trial 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. ;Secondary Objective: ;Primary end point(s): 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. | — |
Countries
United Kingdom
Outcome results
None listed