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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 part a cohort a3: days 1 and 5; part b cohort b3: days 1, 10, and 11
Awards & highlights

Summary

This trial tests a drug to see if it's safe and how it works in the body.

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.Check my eligibility
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.See study design
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 weeks
Treatment ~ Varies
Follow Up ~part a cohort a3: days 1 and 5; part b cohort b3: days 1, 10, and 11
This trial's timeline: 3 weeks for screening, Varies for treatment, and part a cohort a3: days 1 and 5; part b cohort b3: days 1, 10, and 11 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-668
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-668
Part A and B: Urine Concentration of VX-668

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

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.

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Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
247 Previous Clinical Trials
32,489 Total Patients Enrolled

Media Library

VX-668 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05727800 — Phase 1
Alpha-1 Antitrypsin Deficiency Research Study Groups: Part A, Placebo Part A, Part B, Placebo Part B
Alpha-1 Antitrypsin Deficiency Clinical Trial 2023: VX-668 Highlights & Side Effects. Trial Name: NCT05727800 — Phase 1
VX-668 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05727800 — Phase 1
~47 spots leftby Jul 2025