What side effects are associated with this type of intervention?

Common treatments for Alpha-1 Antitrypsin Deficiency, such as augmentation therapy with alpha-1 antitrypsin protein, can have side effects including infusion-related reactions like fever, chills, and headache. Other potential side effects are dizziness, nausea, and fatigue. Rarely, patients may experience severe allergic reactions. These treatments involve intravenous administration, and their pharmacokinetics include rapid absorption into the bloodstream, distribution throughout the body, metabolism primarily in the liver, and excretion via the kidneys.

What prior FDA approvals exist?

Alpha-1 Antitrypsin Deficiency (AATD) treatments approved by the FDA primarily include augmentation therapy with alpha-1 proteinase inhibitors such as Prolastin, Aralast, Zemaira, and Glassia. These therapies are administered intravenously and work by increasing the levels of alpha-1 antitrypsin in the blood to protect the lungs from damage. The pharmacokinetics of these treatments involve absorption directly into the bloodstream, distribution throughout the body, metabolism primarily in the liver, and excretion via the kidneys. While VX-668 is being studied for its safety, tolerability, and pharmacokinetics, it is important to consult with a healthcare provider for the most appropriate treatment options for AATD.

What other types of research exist?

Promising novel alternatives to VX-668 for Alpha-1 Antitrypsin Deficiency patients include drugs that are currently under development or have shown efficacy in related conditions. These may include BMS-663068, a prodrug designed for improved bioavailability, and TAK-272, a renin inhibitor with favorable pharmacokinetics. Both drugs have been selected based on their pharmacokinetic profiles and potential for clinical application. Patients should consult their healthcare provider to discuss these options and determine the best course of treatment.

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VX-668 for Alpha-1 Antitrypsin Deficiency

Phase 1

Waitlist Available

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a new drug called VX-668 to see if it is safe and how the body processes it. The study involves a general population to gather basic safety information.

Who is the study for?

This trial is for adults aged 18-55 with Alpha-1 Antitrypsin Deficiency. They must have a BMI of 18 to 32, weigh over 50 kg, and be non-smokers or haven't smoked for at least three months. Women who can become pregnant are excluded.

What is being tested?

The study tests VX-668 against a placebo to assess its safety, how well it's tolerated by the body, and how the body processes various doses in people with Alpha-1 Antitrypsin Deficiency.

What are the potential side effects?

Possible side effects of VX-668 aren't detailed but typically include reactions at the injection site, gastrointestinal symptoms, headaches, fatigue or allergic responses due to it being first-in-human testing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Part BExperimental Treatment1 Intervention

Participants grouped in different cohorts will receive multiple doses of VX-668.The dose levels will be determined based on the data from Part A.

Group II: Part AExperimental Treatment1 Intervention

Participants grouped in different cohorts will receive a single ascending dose of VX-668.

Group III: Placebo Part APlacebo Group1 Intervention

Participants will be randomized to receive placebo matched to VX-668.

Group IV: Placebo Part BPlacebo Group1 Intervention

Participants will be randomized to receive placebo matched to VX-668.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

VX-668

2023

Completed Phase 1

~120

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatment for Alpha-1 Antitrypsin Deficiency (AATD) is augmentation therapy, which involves regular infusions of alpha-1 antitrypsin protein derived from human plasma. This therapy works by increasing the levels of alpha-1 antitrypsin in the blood and lungs, thereby protecting lung tissue from damage caused by neutrophil elastase. The pharmacokinetics of these treatments—how they are absorbed, distributed, metabolized, and excreted—are critical for maintaining adequate protective levels of the protein in the body. Effective ADME properties ensure that the therapy can consistently provide the necessary protection to prevent lung damage, which is vital for improving the quality of life and health outcomes for AATD patients.

Understanding drug-drug interaction and pharmacogenomic changes in pharmacokinetics for metabolized drugs.Coproporphyrins in Plasma and Urine Can Be Appropriate Clinical Biomarkers to Recapitulate Drug-Drug Interactions Mediated by Organic Anion Transporting Polypeptide Inhibition.Analysis of Intra- and Intersubject Variability in Oral Drug Absorption in Human Bioequivalence Studies of 113 Generic Products.