Phase III, Efficacy and Safety of “Kamada-AAT for Inhalation”
Research type
Research Study
Full title
A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of “Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate Airflow Limitation (50% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%)
IRAS ID
278659
Contact name
Alice Turner
Contact email
Sponsor organisation
Kamada Ltd.
Eudract number
2019-000602-30
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 11 months, 30 days
Research summary
Alpha-1 antitrypsin deficiency (AATD) is a genetic (inherited) condition which affects the body’s ability to produce alpha1-antitrypsin (AAT) protein. AAT protects lung tissue from destruction triggered by inflammation of any cause including smoking and infection. In individuals with AATD, AAT blood level is low and it is less active, so risk of developing a chronic lung disease is high.
Currently AAT treatment is not reimbursed in the UK. Kamada-AAT is approved for intravenous use under the name Glassia® in the USA and other countries. Kamada-AAT for inhalation is the same medicinal product as Glassia® and delivers AAT directly to the lungs using a nebulizer. The advantages of Kamada-AAT for inhalation are:
1. AAT is delivered directly to the lungs, therefore less drug is required as compared to intravenous infusion.
2. Inhalation is simpler and more convenient for patients than intravenous infusion, without the risks associated with infusions.Kamada-AAT for inhalation has been tested in humans
previously; the results from these clinical trials indicate that
Kamada-AAT for inhalation is likely to be safe and welltolerated
in humans.This study will assess the effectiveness and safety of Kamada-AAT for inhalation compared with placebo in patients with AATD.
The study will include approximately 220 participants with AATD.
Duration of participation is approximately 134 weeks including 104 weeks treatment, and 26 weeks follow-up with 14 visits to the study site and 7 telephone calls. Treatment will be blinded, meaning no-one will know which treatment participants are receiving. Participants will undergo procedures such as spirometry, blood samples, questionnaires, and CT scans.
Sponsor: Kamada Ltd.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
20/SC/0181
Date of REC Opinion
2 Jul 2020
REC opinion
Further Information Favourable Opinion