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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03815396




Registration number
NCT03815396
Ethics application status
Date submitted
21/01/2019
Date registered
24/01/2019

Titles & IDs
Public title
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
Scientific 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)
Secondary ID [1] 0 0
Ph1 INBRX-101
Universal Trial Number (UTN)
Trial acronym
rhAAT-Fc
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alpha-1 Antitrypsin Deficiency 0 0
AATD 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - INBRX-101/rhAAT-Fc

Experimental: Part 1 Single Ascending Dose - INBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).

Experimental: Part 2 Multiple Ascending Dose - INBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).


Treatment: Drugs: INBRX-101/rhAAT-Fc
INBRX-101 is a recombinant human alpha-1 antitrypsin (AAT) Fc fusion protein (rhAAT-Fc).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of adverse events of INBRX-101
Timepoint [1] 0 0
Up to 7 months
Primary outcome [2] 0 0
Severity of adverse events of INBRX-101
Timepoint [2] 0 0
Up to 7 months
Secondary outcome [1] 0 0
Area under the serum concentration time curve (AUC) of INBRX-101
Timepoint [1] 0 0
Up to 7 months
Secondary outcome [2] 0 0
Maximum observed serum concentration (Cmax) of INBRX-101
Timepoint [2] 0 0
Up to 7 months
Secondary outcome [3] 0 0
Trough observed serum concentration (Ctrough) of INBRX-101
Timepoint [3] 0 0
Up to 7 months
Secondary outcome [4] 0 0
Time to Cmax (Tmax) of INBRX-101
Timepoint [4] 0 0
Up to 7 months
Secondary outcome [5] 0 0
Half-life (T1/2) of INBRX-101
Timepoint [5] 0 0
Up to 7 months
Secondary outcome [6] 0 0
Immunogenicity of INBRX-101
Timepoint [6] 0 0
Up to 7 months
Secondary outcome [7] 0 0
Distribution of INBRX-101 in Bronchoalveolar Lavage Fluid (BALF)
Timepoint [7] 0 0
Up to 7 months
Secondary outcome [8] 0 0
Functional concentration of INBRX-101 in serum and BALF
Timepoint [8] 0 0
Up to 7 months

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/08/2022
Sample size
Target
Accrual to date
Final
31
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
New Zealand
State/province [5] 0 0
Auckland
Country [6] 0 0
New Zealand
State/province [6] 0 0
Christchurch
Country [7] 0 0
New Zealand
State/province [7] 0 0
Hamilton
Country [8] 0 0
United Kingdom
State/province [8] 0 0
East Of England
Country [9] 0 0
United Kingdom
State/province [9] 0 0
West Midlands

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Inhibrx Biosciences, Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Vasily Andrianov, MD
Address 0 0
Inhibrx Biosciences, Inc
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03815396