Evaluate Efficacy and Safety of "Kamada-AAT for Inhalation" in Patients With AATD
Status:
Recruiting
Trial end date:
2023-05-01
Target enrollment:
Participant gender:
Summary
Alpha-antitrypsin (AAT) is a member of the serpin family of proteinase inhibitors and is to a
large extent responsible for restricting proteinases, notably neutrophil elastase, and
proteinase 3, which might otherwise attack the lung tissue. Individuals with a genetic
deficiency of alpha-1-antitrypsin (AATD) are at a significantly increased risk (80-100%) of
developing emphysema. This study is designed to administer a solution of AAT by nebulizer so
that patients can inhale the drug instead of requiring infusions as in current treatment. A
significant advantage of inhalation is that the AAT is directly transferred to the lungs,
which is the site most in need of the protein. Previous results show that in addition to the
added convenience, three times higher concentrations of AAT can be achieved in the lungs by
inhalation than by intravenous infusions. To date, more than 220 patients have completed
Inhalation studies for several indications.
The current study population will consist of adult patients with congenital alpha-1
antitrypsin (AAT) deficiency who have moderate airflow limitation (forced expiratory volume
in 1 second 50% ≤ [FEV1] ≤ 80% of predicted) and FEV1/slow vital capacity [SVC] ≤ 70% and who
have not experienced two or more moderate or one or more severe exacerbations of COPD during
the past year. A total of 220 patients will be recruited, and after 4 weeks practice inhaling
saline with the nebulizer, will be randomized 1:1 to inhale either 80 mg/day "Kamada-AAT for
Inhalation" or a placebo with identical appearance. Patients will be treated for 104 weeks
and then followed up for a further 26 weeks. Over this time there will be 13 visits to the
clinical study site for evaluation of lung function by blood tests and CT densitometry. In
addition the patients will be required to fill out a daily e-diary with details of the
inhalations and will also report their daily symptoms.