Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

31 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-19

Study Completion Date

2022-08-18

Brief Summary

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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.

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Detailed Description

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Conditions

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Alpha-1 Antitrypsin Deficiency AATD

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part 1 Single Ascending Dose

INBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).

Group Type EXPERIMENTAL

INBRX-101/rhAAT-Fc

Intervention Type DRUG

INBRX-101 is a recombinant human alpha-1 antitrypsin (AAT) Fc fusion protein (rhAAT-Fc).

Part 2 Multiple Ascending Dose

INBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).

Group Type EXPERIMENTAL

INBRX-101/rhAAT-Fc

Intervention Type DRUG

INBRX-101 is a recombinant human alpha-1 antitrypsin (AAT) Fc fusion protein (rhAAT-Fc).

Interventions

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INBRX-101/rhAAT-Fc

INBRX-101 is a recombinant human alpha-1 antitrypsin (AAT) Fc fusion protein (rhAAT-Fc).

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No