Phase 2, single arm, open label study of INBRX-101 in ppt with AATD
Research type
Research Study
Full title
A Phase 2, Single-Arm, Open-Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
IRAS ID
1009912
Contact name
David Kao
Contact email
Sponsor organisation
Inhibrx, Inc.
Clinicaltrials.gov Identifier
Research summary
This is a Phase 2. Multicentre (multiple study sites), single arm (one investigational medicine), open label extension (you and your study doctor will know the medication you receive) study to evaluate the long-term safety and efficacy of the study medication INBRX-101 in patients with alpha-1 antitrypsin deficiency (AATD) emphysema (lung disease, due to damage).
Participants with AATD emphysema have significantly lower blood level of the protein alpha 1 antitrypsin (AAT), AAT has an important function protecting the lungs. INBRX-101 is expected to raise the AAT level in the blood to ‘normal’ physiological levels.
Current A1PI therapies fail to achieve normal levels. INBRX-101 differs from other AATD treatments as it has a special modification designed to prevent loss of the AAT activity over time, meaning the AAT is more stable and so remains in the lungs to function for
longer, creating a clear unmet need for INBRX-101.
There will be 3 cohorts: cohort 1 will consist of participants (ppts) with newly diagnosed AATD emphysema. Cohort 2 will be ppts who completed the parent study, IRAS ID 1008715, and received Zemaira. Cohort 3 will also be ppts who completed the parent study, but received INBRX-101.
INBRX-101 will be administered intravenously (IV, into the vein) every 3 weeks at a dose level of 120 mg/kg. The treatment duration in this study is 3 years. After completing the study treatment, patients will proceed into the safety follow-up phase of the study after the End of Trial Visit.
Assessments will include assessment of vital signs as well as blood/urine tests, electrocardiogram, questionnaires, lung function tests, bronchoscopy (if in cohort 1) and a CT scans.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
24/SC/0191
Date of REC Opinion
30 Jul 2024
REC opinion
Further Information Favourable Opinion