Multi-center, open-label trial to evaluate the safety and tolerability of Alpha-1 MP in subjects with Alpha-1-antitrypsin (a1AT) deficiency. STAMP: Safety and Tolerability of Alpha-1 MP - STAMP

Phase 1

• Conditions: Alpha-1 Antitrypsin (AAT) deficiency; MedDRA version: 9 Level: LLT Classification code 10001806

• Registration Number: EUCTR2005-002402-36-GB
• Lead Sponsor: Talecris Biotherapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2006-02-28
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 35

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 <11uM prior to receiving any augmentation. For naïve subjects, the Alpha 1 Laboratory will also perform a screening Alpha1-Proteinase Inhibitor serum level to determine if the subjects meet this criteria. For those subjects entering the subject that are currently receiving augmentation therapy, historical data obtained at the time the subject was diagnosed with AAT deficiency will be used as verification of meeting this criteria.

3. Documented FEV1 between 20% - 80% of predicted value within last 6 months.

4. Either male or female age = 18 years

5. Written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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;

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified