VX-522 for Cystic Fibrosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Boston Children's Hospital, Boston, MACystic FibrosisVX-522 mRNA therapy - Drug
Eligibility
18 - 65
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions
  • Cystic Fibrosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: From Day 1 Through Safety Follow-up Visit (Week 24)

Week 24
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Trial Safety

Trial Design

1 Treatment Group

VX-522
1 of 1

Experimental Treatment

9 Total Participants · 1 Treatment Group

Primary Treatment: VX-522 · No Placebo Group · Phase 1

VX-522Experimental Group · 2 Interventions: VX-522 mRNA therapy, VX-522 · Intervention Types: Drug, Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from day 1 through safety follow-up visit (week 24)

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
223 Previous Clinical Trials
30,742 Total Patients Enrolled
118 Trials studying Cystic Fibrosis
17,088 Patients Enrolled for Cystic Fibrosis

Eligibility Criteria

Age 18 - 65 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Your body mass index (BMI) is between 18 and 30 kg/m^2.
You have a cystic fibrosis disease that is not getting worse.

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.