A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency

Phase 1

• Conditions: Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency; MedDRA version: 20.0Level: LLTClassification code 10014563Term: Emphysema pulmonarySystem Organ Class: 100000004855; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2013-001870-38-SE
• Lead Sponsor: Grifols Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-09-23
• Last Update Posted: 26 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 339

Inclusion Criteria

1. Have a documented total alpha1-PI serum level < 11 µM.

2. Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or at-risk” alleles. If the genotype has yet to be documented, genotyping (i.e., allelic discrimination) and phenotyping will be performed at the Screening (Week -3) Visit.

3. At the Screening (Week -3) Visit, have a post-bronchodilator FEV1 = 30% and < 80% of
predicted and FEV1/forced vital capacity (FVC) < 70% (Global Initiative for Chronic
Obstructive Lung Disease [GOLD] stage II or III).

4. Have a carbon monoxide diffusing capacity (DLCO) = 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator’s judgment.

5. Have clinical evidence of pulmonary emphysema per the Investigator’s judgment.
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 305
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 34

Exclusion Criteria

1. Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening (Week -3) Visit.
2. Has received alpha1-PI augmentation therapy within one month of the Screening (Week -3) Visit.
3. Has had a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase. Investigator discretion should be used to determine if a subject is appropriate for study participation if the subject has had a COPD exacerbation which occurred more than 5 weeks prior to the Screening (Week -3) Visit.
4. Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
5. History of lung or liver transplant.
6. Any lung surgery during the past 2 years (excluding lung biopsy).
7. On the waiting list for lung surgery, including lung transplant.
8. Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking.
9. History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
10. Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e.,
10 mg every 2 days) within the 5 weeks prior to the Screening (Week -3) Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase. It is recommended to maintain the same dose throughout the study.
11. Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase.
12. Known selective or severe Immunoglobulin A (IgA) deficiency.

Study & Design

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