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mRNA therapy
VX-522 mRNA Therapy for Cystic Fibrosis
Phase 1 & 2
Recruiting
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 older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from day 1 through safety follow-up visit [up to week 24 for sad, and week 28 for t1 and t2 (mad)]
Awards & highlights
Study Summary
This trial looks at how safe and tolerable a drug is for people 18+ with cystic fibrosis that isn't responding to current treatments.
Who is the study for?
This trial is for adults with cystic fibrosis who have specific CFTR gene mutations not treatable with current modulator therapies. Participants must have stable disease, a minimum lung function (FEV1 ≥40%), weigh over 50 kg, and have a BMI under 30. Excluded are those with low oxygen saturation (<94% on room air), uncontrolled asthma in the past year, any organ transplants, or moderate to severe liver issues.Check my eligibility
What is being tested?
The study tests VX-522 mRNA therapy's safety and how well participants tolerate it. It targets individuals aged 18+ whose cystic fibrosis results from certain CFTR gene mutations that don't respond to existing treatments.See study design
What are the potential side effects?
While the side effects of VX-522 are being studied in this trial and aren't fully known yet, common risks may include immune reactions, injection site discomfort, fatigue, fever-like symptoms or potential respiratory complications.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from day 1 through safety follow-up visit [up to week 24 for sad, and week 28 for t1 and t2 (mad)]
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from day 1 through safety follow-up visit [up to week 24 for sad, and week 28 for t1 and t2 (mad)]
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
T1 (MAD): Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
T2 (MAD): Change From Pre-Run-in Baseline in ppFEV1
Trial Design
3Treatment groups
Experimental Treatment
Group I: Single Ascending Dose (SAD)Experimental Treatment1 Intervention
Participants grouped into different cohorts will receive a single ascending dose of VX-522.
Group II: Multiple Ascending Dose (MAD) Arm 1Experimental Treatment1 Intervention
Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1).
Group III: MAD Arm 2: VX522+ IVAExperimental Treatment2 Interventions
Following run-in period with ivacaftor (IVA), participants will receive multiple doses of VX-522 with IVA in treatment arm (T2).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
IVA
2018
Completed Phase 3
~5230
Find a Location
Who is running the clinical trial?
Moderna, IncUNKNOWN
1 Previous Clinical Trials
18 Total Patients Enrolled
Vertex Pharmaceuticals IncorporatedLead Sponsor
243 Previous Clinical Trials
32,351 Total Patients Enrolled
126 Trials studying Cystic Fibrosis
17,713 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your cystic fibrosis (CF) is not getting worse.Your lung function test results show that your breathing is less than 40% of what is expected for someone your age, sex, and height.Your blood oxygen level is less than 94% when breathing normal air.You have had uncontrolled asthma in the past year before the screening.Your body mass index is less than 30.You have had a solid organ or bone marrow transplant in the past.You weigh more than 50 kilograms.You have liver cirrhosis with portal hypertension or moderate to severe liver impairment based on a scoring system called Child Pugh Score.You have specific mutations in both copies of your CFTR gene that do not respond to a certain type of therapy. These mutations stop the CFTR protein from working properly.
Research Study Groups:
This trial has the following groups:- Group 1: Single Ascending Dose (SAD)
- Group 2: MAD Arm 2: VX522+ IVA
- Group 3: Multiple Ascending Dose (MAD) Arm 1
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- 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
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has the federal agency approved VX-522 for medical use?
"Our team has assigned VX-522 a score of 1 on the safety scale since it is still in its first phase and there is limited evidence supporting both efficacy and safety."
Answered by AI
How many patients have enrolled in the research thus far?
"Aye, according to clinicaltrials.gov's records, this medical research is actively welcoming applicants. The initial posting was on January 1st of 2023 and the most recent edit took place a few months later on April 1st. This study requires 9 patients from a single site."
Answered by AI
Do I fulfill the requirements to join this medical research project?
"Aspiring participants of this trial need to have a diagnosis of cystic fibrosis and be between 18-65 years old. Currently, the team is looking for approximately 9 individuals who meet these criteria."
Answered by AI
Is this research open to minors?
"This clinical trial only accepts participants aged 18-65 years old. However, there are a total of 85 trials designed for minors and 371 trials that accommodate elderly patients."
Answered by AI
Are there any openings left for individuals to take part in this clinical trial?
"Affirmative. The clinical trial details listed on the clinicaltrials.gov website corroborate that this experiment is presently recruiting participants; it was initially posted on January 1st 2023 and updated as recently as April 1st, 2023. At present, 9 volunteers are needed at a single medical facility for participation in the study."
Answered by AI
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