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Corrector

VX-864 for Alpha-1 Antitrypsin Deficiency

Phase 2
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
Participants must have a PiZZ genotype confirmed at screening
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 up to week 52
Awards & highlights

Study Summary

This trial looks at how safe and effective a drug is for people with a specific gene mutation over 48 weeks.

Who is the study for?
This trial is for individuals with severe Alpha-1 Antitrypsin Deficiency, confirmed by low plasma AAT levels and a PiZZ genotype. It's not suitable for those with conditions that may interfere with study completion, who can't stop augmentation therapy, have had gene or RNAi therapies before, or are on a transplant list.Check my eligibility
What is being tested?
The trial is testing the effectiveness and safety of a drug called VX-864 in participants over a period of 48 weeks. The focus is on people with the specific PiZZ genotype associated with Alpha-1 Antitrypsin Deficiency.See study design
What are the potential side effects?
While specific side effects of VX-864 aren't listed here, common ones could include reactions at the injection site, liver issues due to increased AAT production, fatigue, headaches or gastrointestinal discomfort.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My genetic test shows I have the PiZZ genotype.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 up to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 up to week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels
Secondary outcome measures
Change in Blood Antigenic AAT Levels
Change in Blood Functional AAT Levels
Change in Blood Z-polymer Levels
+2 more

Side effects data

From 2021 Phase 2 trial • 44 Patients • NCT04474197
20%
Fatigue
20%
Pyrexia
20%
Cough
10%
Dizziness
10%
Nasal congestion
10%
Dyspnoea
10%
Vaginal haemorrhage
10%
Haemoglobin increased
10%
Toothache
10%
Headache
10%
Feeling abnormal
10%
Sputum increased
10%
Tachypnoea
10%
Rash
10%
Diarrhoea
10%
Abdominal discomfort
10%
Abdominal pain
10%
Feeling cold
10%
Urinary tract infection
10%
Oxygen saturation decreased
10%
Respiration abnormal
10%
Aspartate aminotransferase increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
VX-864 100 mg
VX-864 300 mg
VX-864 500 mg

Trial Design

2Treatment groups
Experimental Treatment
Group I: Group BExperimental Treatment1 Intervention
Participants will undergo a liver biopsy done before receiving VX-864 q12h for 48 weeks, and will undergo a second liver biopsy at either Week 24 or Week 48.
Group II: Group AExperimental Treatment1 Intervention
Participants will receive VX-864 every 12 hours (q12h) for 48 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
VX-864
2020
Completed Phase 2
~50

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
241 Previous Clinical Trials
32,221 Total Patients Enrolled

Media Library

VX-864 (Corrector) Clinical Trial Eligibility Overview. Trial Name: NCT05643495 — Phase 2
Alpha-1 Antitrypsin Deficiency Research Study Groups: Group A, Group B
Alpha-1 Antitrypsin Deficiency Clinical Trial 2023: VX-864 Highlights & Side Effects. Trial Name: NCT05643495 — Phase 2
VX-864 (Corrector) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05643495 — Phase 2

Frequently Asked Questions

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

How hazardous is exposure to Group A for individuals?

"Despite limited evidence of efficacy, Group A has sufficient data to support its safety rating of 2."

Answered by AI

Does the protocol of this clinical trial encompass individuals over fifty years old?

"This trial is open to patients aged 18-80. There are also additional clinical trials for minors and seniors, with 1 study specifically targeting those under the age of 18 and 12 studies tailored to people over 65 years old."

Answered by AI

Is this research actively seeking participants?

"Affirmative. According to clinicaltrials.gov, this scientific investigation is still seeking participants; it was initially announced on December 1st 2022 and recently updated on the 22nd of that same month. The trial necessitates 20 enrollees from two separate locations."

Answered by AI

What is the current capacity for participants in this trial?

"Affirmative. According to the data found on clinicaltrials.gov, this study is presently recruiting test subjects. It was first posted on December 1st 2022 and has most recently been updated on December 22nd 2022 with a goal of finding 20 people across 2 sites."

Answered by AI

What criteria must be fulfilled to qualify for this research project?

"This investigation is including 20 individuals with alpha 1-antitrypsin ranging from the age of 18 to 80. Most crucially, applicants must demonstrate a PiZZ genotype during screening and have insufficient AAT levels in their plasma."

Answered by AI
~7 spots leftby Nov 2024