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VX-864 for Alpha-1 Antitrypsin Deficiency
Study Summary
This trial looks at how safe and effective a drug is for people with a specific gene mutation over 48 weeks.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2021 Phase 2 trial • 44 Patients • NCT04474197Trial Design
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Who is running the clinical trial?
Media Library
- My doctor thinks stopping my current treatment isn't good for me.I have never undergone gene therapy or RNA interference therapy.Your blood test shows a severe lack of AAT.My genetic test shows I have the PiZZ genotype.I have had an organ or bone marrow transplant or am on a waiting list for one.
- Group 1: Group A
- Group 2: Group B
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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."
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."
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