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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 weeks
Treatment ~ Varies
Follow Up ~from baseline through week 96
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline through week 96 for reporting.

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 • NCT03068312
13%
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
243 Previous Clinical Trials
32,301 Total Patients Enrolled
126 Trials studying Cystic Fibrosis
17,663 Patients Enrolled for Cystic Fibrosis

Media Library

Ivacaftor (CFTR Potentiator) Clinical Trial Eligibility Overview. Trial Name: NCT03277196 — Phase 3
Cystic Fibrosis Research Study Groups: Ivacaftor Arm, Observational Arm
Cystic Fibrosis Clinical Trial 2023: Ivacaftor Highlights & Side Effects. Trial Name: NCT03277196 — Phase 3
Ivacaftor (CFTR Potentiator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03277196 — Phase 3

Frequently Asked Questions

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

Is this a groundbreaking clinical trial?

"Ivacaftor has been under scientific scrutiny since 2015 when the first clinical trial, sponsored by Vertex Pharmaceuticals Incorporated, took place. This initial study included 1044 participants. After this Phase 3 drug approval was received in 2015, there have been 22 active trials for Ivacaftor across 110 cities and 23 countries."

Answered by AI

What is the maximum sample size for this research?

"Presently, this trial is not actively recruiting patients. The lastest update to the study was on September 14th, 2022 and it was first posted on August 16th, 2017. If you are looking for other studies involving cystic fibrosis or Ivacaftor, there are currently 432 clinical trials enrolling patients and 22 studies respectively that are actively searching for participants."

Answered by AI

If I wanted to, could I join this experiment?

"This particular clinical trial is testing a medication for cystic fibrosis in infants aged 0-24 months. A total of 86 patients will be enrolled in the study."

Answered by AI

Could you tell me how many different places this trial is being administered?

"Currently, this study is being conducted at 19 sites located in Seattle, Houston, Billings and 16 other cities. If you wish to enroll, please choose the location nearest to you in order to reduce travel costs and time."

Answered by AI

Are patients of all ages being accepted for this clinical trial?

"This study is looking for children aged 0 to 24 months old."

Answered by AI

Is Ivacaftor a common medication in research?

"There are a total of 22 different ongoing clinical trials for Ivacaftor, 15 of which have reached Phase 3. The majority of these studies originate from Bochum and California; however, there are 1,038 unique locations conducting research on Ivacaftor."

Answered by AI

What are the main conditions that Ivacaftor is used to treat?

"Ivacaftor is an effective medication for patients that have the F508del mutation in the CFTR gene and have been diagnosed with cystic fibrosis."

Answered by AI

Can new patients still sign up for this experiment?

"The listing for this trial on clinicaltrials.gov says that it is not recruiting patients at the moment. The study was initially posted on August 16th, 2017 and updated September 14th, 2020. 454 other trials are presently looking for participants."

Answered by AI

Has Ivacaftor received clearance from the FDA?

"Ivacaftor received a safety score of 3 because multiple rounds of clinical data support its efficacy and safety."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
2017. "A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03277196.
~11 spots leftby Apr 2025
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