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CFTR Potentiator

Part A: 3 to <24 months for Cystic Fibrosis

Q: What medical condition does ivacaftor usually treat?

Patients with the F508del mutation in the CFTR gene can be treated for <a href="https://www.withpower.com/clinical-trials/cystic-fibrosis">cystic fibrosis</a> by taking ivacaftor.

Q: Does this research break new ground in the field?

There are 22 clinical trials involving ivacaftor that are ongoing as of now. They span 110 cities and 23 countries, with the first one having been run in 2015. The sponsor for this earliest study was Vertex Pharmaceuticals Incorporated--1044 people participated and it completed its Phase 3 drug approval stage. In the 5 years since then, 85 more studies have wrapped up.

Q: Is this research project open to new participants at the moment?

As of 8/30/2022, this clinical trial is no longer recruiting patients. However, there are 432 other trials for <a href="https://www.withpower.com/clinical-trials/cystic-fibrosis">cystic fibrosis</a> and 22 trials for ivacaftor that are still searching for participants.

Q: How many people are volunteering to participate in this experiment?

This study has completed patient recruitment for the trial. The first posting was on March 1st, 2016 and the most recent update was on August 30th, 2022. If you are looking for other studies to participate in, there are currently 432 trials searching for patients with <a href="https://www.withpower.com/clinical-trials/cystic-fibrosis">cystic fibrosis</a> and 22 trials searching for patients that will test ivacaftor.

Q: Has the FDA cleared ivacaftor for use?

There is some evidence from previous trials that ivacaftor is effective, and it has undergone multiple rounds of testing to confirm its safety, so it received a score of 3.

Q: Are patients above the age of 45 still being recruited for this research project?

For this particular trial, subjects must be infants aged 0 to 24 months old. Out of the 508 total trials, 111 are for patients under 18 years old and 407 are for individuals above 65 years old.

Q: How many different hospitals are conducting this research?

Enrolment for this program is open at 14 discrepant locations; which include major cities such as Chicago, Baltimore and Philadelphia. To reduce the strain of travelling, it is recommended that you enroll at a location closest to you.

Q: Could you please summarize the research conducted on ivacaftor to date?

At this moment, there are 22 ivacaftor trials ongoing with 15 in the critical Phase 3. Although a large portion of these research studies are situated in Bochum and <a href="https://www.withpower.com/clinical-trials/california">California</a>, there are 1038 clinical trial sites for ivacaftor globally.

Q: Am I eligible to participate in this research project?

Up to 56 <a href="https://www.withpower.com/clinical-trials/cystic-fibrosis">cystic fibrosis</a> patients aged 0-24 months can enroll in this trial. To be eligible, they must have one of the following ten CFTR mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P,G1349D or R117H. Part A/B group may also have other ivacaftor-responsive mutations..

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 day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); day 15 (pre-dose); week 4 (pre-dose); week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); week 12 (pre-dose); week 18 (pre-dose) and week 24 (pre-dose)

Awards & highlights

Study Summary

This trial will study the safety and how well ivacaftor works in treating CF in children less than 24 months old with a certain gene mutation.

Eligible Conditions

Cystic Fibrosis

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); day 15 (pre-dose); week 4 (pre-dose); week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); week 12 (pre-dose); week 18 (pre-dose) and week 24 (pre-dose)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); day 15 (pre-dose); week 4 (pre-dose); week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); week 12 (pre-dose); week 18 (pre-dose) and week 24 (pre-dose)

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); day 15 (pre-dose); week 4 (pre-dose); week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); week 12 (pre-dose); week 18 (pre-dose) and week 24 (pre-dose) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part A/B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)

Part A: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)

+1 more

Secondary outcome measures

Part B + A/B: Absolute Change From Baseline in Sweat Chloride

Part B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)

Side effects data

From 2022 Phase 3 trial • 458 Patients • NCT04043806

25%

Cough

23%

Infective pulmonary exacerbation of cystic fibrosis

20%

Headache

16%

Upper respiratory tract infection

15%

Sputum increased

14%

Pyrexia

13%

Oropharyngeal pain

13%

Nasopharyngitis

11%

Nasal congestion

10%

Fatigue

10%

Viral upper respiratory tract infection

Nausea

Immunisation reaction

Alanine aminotransferase increased

Blood creatine phosphokinase increased

Haemoptysis

Rhinorrhoea

Abdominal pain

Diarrhoea

COVID-19

Sinusitis

Aspartate aminotransferase increased

Back pain

Arthralgia

Dyspnoea

Productive cough

Sinus congestion

Rash

Vomiting

Pain

Myalgia

Respiration abnormal

Pneumonia

Distal intestinal obstruction syndrome

100%

80%

60%

40%

20%

Study treatment Arm

Part A: ELX/TEZ/IVA

Part B: ELX/TEZ/IVA

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part B + A/B:1 to < 24 monthsExperimental Treatment1 Intervention

Participants 4 to <6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to <7 kg received 25 mg IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to <4 months weighing 3 kg to <5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.

Group II: Part A: 3 to <24 monthsExperimental Treatment1 Intervention

Participants weighing 5 to less than (<) 7 kilogram (kg) received 25 milligram (mg) IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

IVA

2018

Completed Phase 3

~5230

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,328 Total Patients Enrolled

126 Trials studying Cystic Fibrosis

17,692 Patients Enrolled for Cystic Fibrosis

Frequently Asked Questions

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

What medical condition does ivacaftor usually treat?

"Patients with the F508del mutation in the CFTR gene can be treated for cystic fibrosis by taking ivacaftor."

Answered by AI

Does this research break new ground in the field?

"There are 22 clinical trials involving ivacaftor that are ongoing as of now. They span 110 cities and 23 countries, with the first one having been run in 2015. The sponsor for this earliest study was Vertex Pharmaceuticals Incorporated--1044 people participated and it completed its Phase 3 drug approval stage. In the 5 years since then, 85 more studies have wrapped up."

Answered by AI

Is this research project open to new participants at the moment?

"As of 8/30/2022, this clinical trial is no longer recruiting patients. However, there are 432 other trials for cystic fibrosis and 22 trials for ivacaftor that are still searching for participants."

Answered by AI

How many people are volunteering to participate in this experiment?

"This study has completed patient recruitment for the trial. The first posting was on March 1st, 2016 and the most recent update was on August 30th, 2022. If you are looking for other studies to participate in, there are currently 432 trials searching for patients with cystic fibrosis and 22 trials searching for patients that will test ivacaftor."

Answered by AI

Has the FDA cleared ivacaftor for use?

"There is some evidence from previous trials that ivacaftor is effective, and it has undergone multiple rounds of testing to confirm its safety, so it received a score of 3."

Answered by AI

Are patients above the age of 45 still being recruited for this research project?

"For this particular trial, subjects must be infants aged 0 to 24 months old. Out of the 508 total trials, 111 are for patients under 18 years old and 407 are for individuals above 65 years old."

Answered by AI

How many different hospitals are conducting this research?

"Enrolment for this program is open at 14 discrepant locations; which include major cities such as Chicago, Baltimore and Philadelphia. To reduce the strain of travelling, it is recommended that you enroll at a location closest to you."

Answered by AI

Could you please summarize the research conducted on ivacaftor to date?

"At this moment, there are 22 ivacaftor trials ongoing with 15 in the critical Phase 3. Although a large portion of these research studies are situated in Bochum and California, there are 1038 clinical trial sites for ivacaftor globally."

Answered by AI

Am I eligible to participate in this research project?

"Up to 56 cystic fibrosis patients aged 0-24 months can enroll in this trial. To be eligible, they must have one of the following ten CFTR mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P,G1349D or R117H. Part A/B group may also have other ivacaftor-responsive mutations.."

Answered by AI

Recent research and studies

The Lancet Respiratory MedicineJournal

Ivacaftor Treatment of Cystic Fibrosis in Children Aged 12 to <24 Months and with a CFTR Gating Mutation (ARRIVAL): A Phase 3 Single-arm Study

Rosenfeld, Margaret, Claire E Wainwright, Mark Higgins, Linda T Wang, Charlotte McKee, Daniel Campbell, Simon Tian, et al.. 2018. “Ivacaftor Treatment of Cystic Fibrosis in Children Aged 12 to The Lancet Respiratory Medicine. Elsevier BV. doi:10.1016/s2213-2600(18)30202-9.

American Journal of Respiratory and Critical Care MedicineJournal

Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-part Phase 3 Clinical Trial

Davies, Jane C., Claire E. Wainwright, Gregory S. Sawicki, Mark N. Higgins, Daniel Campbell, Christopher Harris, Paul Panorchan, Eric Haseltine, Simon Tian, and Margaret Rosenfeld. 2021. “Ivacaftor in Infants Aged 4 to American Journal of Respiratory and Critical Care Medicine. American Thoracic Society. doi:10.1164/rccm.202008-3177oc.

clinicaltrials.govStudy

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

2016. "A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02725567.