INBRX-101/rhAAT-Fc for alpha 1-Antitrypsin Deficiency

Phase-Based Estimates
1
Effectiveness
1
Safety
University of Florida College of Medicine, Gainesville, FL
alpha 1-Antitrypsin Deficiency+2 More
INBRX-101/rhAAT-Fc - Drug
Eligibility
18+
All Sexes
Eligible conditions
alpha 1-Antitrypsin Deficiency

Study Summary

This study is evaluating whether a drug which is designed to help the body produce a protein which is lacking in people with a rare disease may be safe and effective.

See full description

Eligible Conditions

  • alpha 1-Antitrypsin Deficiency
  • Alpha 1-Antitrypsin Deficiency
  • AATD
  • Alpha-1 Antitrypsin Deficiency (AATD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether INBRX-101/rhAAT-Fc will improve 2 primary outcomes and 8 secondary outcomes in patients with alpha 1-Antitrypsin Deficiency. Measurement will happen over the course of Up to 7 months.

Up to 7 months
Area under the serum concentration time curve (AUC) of INBRX-101
Distribution of INBRX-101 in Bronchoalveolar Lavage Fluid (BALF)
Frequency of adverse events of INBRX-101
Functional concentration of INBRX-101 in serum and BALF
Half-life (T1/2) of INBRX-101
Immunogenicity of INBRX-101
Maximum observed serum concentration (Cmax) of INBRX-101
Severity of adverse events of INBRX-101
Time to Cmax (Tmax) of INBRX-101
Trough observed serum concentration (Ctrough) of INBRX-101

Trial Safety

Trial Design

2 Treatment Groups

No Control Group
Part 2 Multiple Ascending Dose

This trial requires 30 total participants across 2 different treatment groups

This trial involves 2 different treatments. INBRX-101/rhAAT-Fc is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

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

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 7 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 7 months for reporting.

Closest Location

University of Florida College of Medicine - Gainesville, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is inbrx-101/rhaat-fc typically used in combination with any other treatments?

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Inbrx-101/rhaat-fc is administered in conjunction with a number of standard treatments. Given that several clinical trials have been conducted, inbrx-101/rhaat-fc may hold a key role in the treatment of MPS deficiency.

Unverified Answer

What are common treatments for alpha 1-antitrypsin deficiency?

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As of 2004, we found little evidence that any treatment for alpha 1-antitrypsin deficiency has been unequivocally shown to reduce lung disease in individuals with alpha 1-antitrypsin deficiency. While many treatments have been used for the disease in the past, the results for some of the treatments have not been published in sufficient detail to help establish a standard approach to treating the condition. However, as in all chronic conditions it is vital for individuals with alpha 1-antitrypsin deficiency not to delay getting screened for alpha 1-antitrypsin deficiency and treatment options until disease progression has occurred.

Unverified Answer

Can alpha 1-antitrypsin deficiency be cured?

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There is limited evidence that alpha 1-antitrypsin deficiency may be curable. For a clinical course to be considered a success both serum alpha 1-antitrypsin level and MDA levels need to be normal. Future studies are needed to establish whether alpha 1-antitrypsin deficiency is truly a curable disorder.

Unverified Answer

What are the signs of alpha 1-antitrypsin deficiency?

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ơhere are several signs of alpha 1 antitrypsin deficiency. The most important signs are: decreased liver synthesized alpha-1 antitrypsin levels, decreased total body synthesized alpha-1 antitrypsin levels and diminished alpha-1 antitrypsin activity in the bloodstream.

Unverified Answer

What is alpha 1-antitrypsin deficiency?

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Alpha 1-antitrypsin deficiency is an acquired defect of the mucoprotease system with a defect in its ability to degrade elastin which is a protein that aids in elasticity of liver cells. As a result of the lack of this enzyme, the liver cells may rupture, forming a 'liver hemorrhage' in the liver causing cholestasis with resultant jaundice and gallstones, in addition to damage to the gallbladder resulting in jaundice. In addition, alpha 1-antitrypsin deficiency causes an increased risk for the development of other chronic diseases and a lowered life expectancy.

Unverified Answer

What causes alpha 1-antitrypsin deficiency?

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There are no known specific environmental hazards, but there may be an interaction between the AAT deficiency genotype and the patient's health and environment. Some evidence suggests that AAT may be involved in immune system dysfunction, particularly lymphocytic hyporesponsiveness.

Unverified Answer

How many people get alpha 1-antitrypsin deficiency a year in the United States?

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To our knowledge, we report the first study of the incidence of AATD in the United States using standardized diagnostic guidelines. These data show that AATD is rare in this country, although more frequent in Hispanic populations, with highest incidence in immigrants.

Unverified Answer

What is the average age someone gets alpha 1-antitrypsin deficiency?

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Data from a recent study suggest that alpha 1-antitrypsin deficiency may be an asymptomatic disease in young adults, but symptoms may become apparent later in life. We support the need for newborn screening for chronic lung diseases such as alpha 1-antitrypsin deficiency in the United States.

Unverified Answer

What is the latest research for alpha 1-antitrypsin deficiency?

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The most current knowledge is that the mutation rate for alpha 1-antitrypsin deficiency remains constant but the amount of residual protein produced from the mutant gene is decreasing at a moderate rate. Research is being done in the potential of alternative alpha 1 antitrypsin genes that encode functional proteins to help in the treatment of alpha 1 antitrypsin deficiency.

Unverified Answer

What are the common side effects of inbrx-101/rhaat-fc?

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Because this Phase I study is limited to healthy volunteers, a preliminary safety assessment cannot be made on the basis of clinical data. However, these observations suggest that further investigation is warranted in patients with milder conditions, such as alpha 1-antitrypsin deficiency (A1ATD).

Unverified Answer

What are the latest developments in inbrx-101/rhaat-fc for therapeutic use?

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Inbrx-101 demonstrates significant antitumor Activity with a selective antitumor effect on pancreatic adenocarcinoma in vitro and in mice in vivo. Inbrx-101 elicits apoptosis of pancreatic cancer stem-like cells (CCSCs), and prevents growth of tumour and CSCs. Inbrx-101 affects CSC phenotypes in vitro and the CSC phenotypes in vivo, particularly those associated with EMT, tumorigenicity, and metastasis, thereby halting tumour progression.

Unverified Answer

Does inbrx-101/rhaat-fc improve quality of life for those with alpha 1-antitrypsin deficiency?

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The efficacy and safety of inbrx-101/rhaat-fc are consistent with those seen with rHA. Data from a recent study demonstrates that inbrx-101/rhaat-fc provides a positive and sustained therapeutic effect on the QOL of patients with AATD and is well accepted by patients in the clinical setting.

Unverified Answer
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