Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1
will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will
consist of multiple ascending dose (MAD) administrations of INBRX-101. The planned dosing
schedule is IV every 3 to 4 weeks.
Phase
Phase 1
Funding Agency/Sponsor
Industry
Disease
Other
Enrollment Eligibility
Inclusion Criteria:
- Documented alpha-1 antitrypsin (AAT) serum concentration <11 μM.
- Diagnosis of alpha-1 antitrypsin deficiency (AATD) with any allelic combination with
exception of the null/null genotype.
- For subjects in Part 2 80 and 120 mg/kg cohorts ONLY: post-bronchodilator FEV1 of at
least 40% of predicted normal value.
- For subjects in Part 2 80 and 120 mg/kg cohorts ONLY: subjects eligible for
bronchoscopy per judgment of investigator.
- Nonsmoker for at least 6 months prior to study and must remain nonsmoking for the
entire study duration.
- Adequate hepatic and renal function as defined per protocol.
- Willing to undergo current augmentation therapy washout (if applicable) and refrain
from initiating augmentation therapy, other investigational drug trials for AATD,
therapy with IV immunoglobulins or monoclonal antibodies during the entire study,
including follow-up.
Exclusion Criteria:
- Known or suspected allergy to components of INBRX-101 (AAT or human IgG) or pdAAT.
- Participation in any investigational drug trial within 30 days prior to this trial, or
subjects receiving IV immunoglobulins or monoclonal antibodies within 30 days prior to
this trial.
- History of and/or on the waiting list for lung or liver transplant, lobectomy, or lung
volume reduction surgery.
- Acute respiratory tract infection or COPD exacerbation that required antibiotic
treatment and/or increase in systemic steroid dosage within the 4 weeks prior to
screening. Subjects are permitted to continue to receive steroids if the investigator
judges the subject to have a history of stable dosing.
- Subjects with ongoing or history of unstable cor pulmonale.
- Infection with hepatitis A, B, or C or human immunodeficiency virus (HIV).
- Active autoimmune disease or documented history of autoimmune disease that 1) required
systemic steroids or immune-suppressive medications and 2) tested positive for
auto-antibodies. Exception: Endocrinopathies managed with hormone replacement therapy
(HRT).
- Current substance and/or alcohol abuse with protocol defined exceptions.
- Current narcotics abuse with protocol defined exceptions.