Brief Title
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
Official Title
An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Brief 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.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Frequency of adverse events of INBRX-101
Secondary Outcome
Area under the serum concentration time curve (AUC) of INBRX-101
Condition
Alpha-1 Antitrypsin Deficiency
Intervention
INBRX-101/rhAAT-Fc
Study Arms / Comparison Groups
Part 1 Single Ascending Dose
Description: INBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
30
Start Date
July 19, 2019
Completion Date
December 2021
Primary Completion Date
December 2021
Eligibility Criteria
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.
Gender
All
Ages
18 Years - 80 Years
Accepts Healthy Volunteers
No
Contacts
Klaus Wagner, MD, PhD, ,
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT03815396
Organization ID
Ph1 INBRX-101
Responsible Party
Sponsor
Study Sponsor
Inhibrx, Inc.
Study Sponsor
Klaus Wagner, MD, PhD, Study Director, CMO
Verification Date
May 2020