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CFTR Potentiator
Long-term Ivacaftor Treatment for Cystic Fibrosis
Phase 3
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 younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline through week 96
Awards & highlights
Study Summary
This trial is testing the safety and effectiveness of a drug called ivacaftor in treating cystic fibrosis in children under 2 years old.
Who is the study for?
This trial is for children with cystic fibrosis under 24 months old who have a specific mutation responsive to Ivacaftor. They can join if they've completed Study 124 Part B or are newly diagnosed, provided their parents understand the study and consent. Those with conditions that could risk safety or skew results, recent infections, abnormal liver function, low hemoglobin levels, organ transplants, or recent use of certain other drugs cannot participate.Check my eligibility
What is being tested?
The study tests long-term safety and effectiveness of Ivacaftor in young children with cystic fibrosis. It's a Phase 3 trial with two groups: one receiving Ivacaftor (open-label) and an observational group not currently on treatment but monitored due to previous participation in Study 124 Part B.See study design
What are the potential side effects?
While not specified here, common side effects of Ivacaftor may include headache, upper respiratory tract infection (like colds), stomach pain, diarrhea, rash and dizziness. The exact side effects will be closely monitored throughout the trial.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from baseline through week 96
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline through week 96
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Secondary outcome measures
Absolute change in sweat chloride
Side effects data
From 2018 Phase 3 trial • 38 Patients • NCT0306831213%
Infective pulmonary exacerbation of cystic fibrosis
8%
Haemoptysis
8%
Upper respiratory tract infection
5%
Pyrexia
3%
Abortion spontaneous
3%
Headache
3%
Viral upper respiratory tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Ivacaftor
Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: Ivacaftor ArmExperimental Treatment1 Intervention
Group II: Observational ArmActive Control1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ivacaftor
2013
Completed Phase 3
~2950
Find a Location
Who is running the clinical trial?
Vertex Pharmaceuticals IncorporatedLead Sponsor
246 Previous Clinical Trials
32,496 Total Patients Enrolled
126 Trials studying Cystic Fibrosis
17,666 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a medical condition that could affect the study results or make it risky for you to take ivacaftor.You are currently being treated with ivacaftor.You have been diagnosed with cystic fibrosis, have specific genetic mutations related to cystic fibrosis, and live in an area where ivacaftor is approved for use in people over 2 years old. Your parent or legal guardian must understand and agree to follow the study rules.Criterion: You have a medical condition or illness that could affect the study results or may increase the risk of taking ivacaftor. You have had a recent respiratory infection or changes in pulmonary disease treatment, abnormal liver function, low hemoglobin levels, or a history of organ or blood transplants. You have also taken certain medications within two weeks of the study start date.You were in a previous study where you took ivacaftor, and you chose not to join this study or are not eligible to join the part of this study that involves taking ivacaftor.
Research Study Groups:
This trial has the following groups:- Group 1: Ivacaftor Arm
- Group 2: Observational Arm
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
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.
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