A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema

Kamada, Ltd.12 sites in 7 countries168 target enrollmentDecember 2009

ConditionsEmphysema

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Emphysema
Sponsor: Kamada, Ltd.
Enrollment: 168
Locations: 12
Primary Endpoint: Exacerbation events and lung density
Status: Completed
Last Updated: 6 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This is a randomised , placebo controlled, double blind , multicentre, Phase II/III study evaluating the safety and efficacy of Kamada AAT for inhalation in patients with Emphysema caused by Alpha-1 Antitrypsin (AAT) deficiency.

Detailed Description

Alpha-1 Antitrypsin Deficiency, also called Alpha-1-Proteinase Inhibitor (API) deficiency, is a genetic disorder characterized by the production of an abnormal amount of AAT protein and reduced circulating levels of this protein. Subjects with AAT deficiency are at increased risk for developing Emphysema. It is believed that this is the result of the chronic activity of elastase released by cells continually present in the lungs in low numbers. Three blinded interim analyses have shown that there are no safety issues and no concerns regarding tolerability.

Registry

clinicaltrials.gov

Start Date

December 2009

End Date

June 2015

Last Updated

6 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Kamada, Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Diagnosis of emphysema confirmed by CT scan. If a report of past CT scan is not available at site documenting then a CT scan is to be performed at screening
•Male or female patients at least 18 years of age.
•Able and willing to sign an informed consent.
•Patient with record of congenital AAT deficiency of phenotype PiZZ (homozygote) or other rare phenotypes related to AAT deficiency and with AAT serum level ≤ 11 micromole. For patients receiving IV AAT augmentation therapy the serum AAT level threshold does not apply.
•FEV1/SVC \<70% of predicted value post bronchodilator (SVC is slow VC) and FEV1 \< 80% of predicted value post-bronchodilator
•History of at least two moderate or severe exacerbations that required change in treatment (antibiotics, systemic steroids, hospitalization) in the last 18 months prior to date of screening , with at least one of these occurring within the last 12 months prior to screening.
•Ability to comply with completion of electronic diary.
•Ability to self-administer inhaled AAT.
•No significant abnormalities in serum hematology, serum chemistry and serum inflammatory / immunogenic markers according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.
•No significant abnormalities in urinalysis according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.

Exclusion Criteria

•FEV1 \>= 80% or FEV1 \< 20% of predicted value post-bronchodilator.
•FEV1/SVC\>=70%
•History of lung transplant.
•Any lung surgery within the past two years.
•On any thoracic surgery waiting list.
•End of last exacerbation less than 6 weeks prior to screening/re-screening visit.
•Clinically significant intercurrent illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal or other) that in the opinion of the investigator, could interfere with the safety, compliance or other aspects of this study. Patients with well-controlled, chronic diseases could possibly be included after consultation with the treating physician and the sponsor.
•Active smoking during the last 12 months from screening date.
•Pregnancy or lactation.
•Woman of child-bearing potential not taking adequate contraception deemed reliable by the investigator.