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Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator

ELX/TEZ/IVA for Cystic Fibrosis

Phase 3

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

Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 15 up to week 16

Awards & highlights

Study Summary

This trial will study how drugs work in young CF patients, to see if they are safe and effective.

Who is the study for?

This trial is for young children aged 12 to less than 24 months with Cystic Fibrosis who have at least one F508del mutation or another mutation responsive to the study drugs. Children should not have other illnesses that could confuse the results or increase risks from the study drugs.Check my eligibility

What is being tested?

The trial is testing ELX/TEZ/IVA, a combination of medications for Cystic Fibrosis, in very young children. It aims to understand how this medication works in their bodies and its safety, tolerability, and effectiveness.See study design

What are the potential side effects?

While specific side effects are not listed here, common ones may include digestive issues, liver problems, respiratory symptoms like coughing or wheezing; each child's experience can vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have a specific mutation in my CF gene that responds to certain treatments.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 15 up to week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 15 up to week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 15 up to week 16 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: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites

Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary outcome measures

Part B: Absolute Change in Sweat Chloride (SwCl)

Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part BExperimental Treatment2 Interventions

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.

Group II: Part AExperimental Treatment2 Interventions

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ELX/TEZ/IVA

2019

Completed Phase 3

~3370

IVA

2018

Completed Phase 3

~5230

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,323 Total Patients Enrolled

126 Trials studying Cystic Fibrosis

17,685 Patients Enrolled for Cystic Fibrosis

Media Library

ELX/TEZ/IVA (Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT05882357 — Phase 3

Cystic Fibrosis Research Study Groups: Part A, Part B

Cystic Fibrosis Clinical Trial 2023: ELX/TEZ/IVA Highlights & Side Effects. Trial Name: NCT05882357 — Phase 3

ELX/TEZ/IVA (Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05882357 — Phase 3

Frequently Asked Questions

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

Does this clinical investigation accept members who are octogenarian or older?

"This research is enrolling participants who are between 12 Months and 24 Months old."

Answered by AI

Are individuals still being enrolled in this experiment?

"Affirmative. Clinicaltrials.gov has a record of this medical trial that was first made available on June 27, 2023 and was recently updated in July 14th, 2023. They are hoping to enrol 64 patients from two distinct clinical locations."

Answered by AI

How hazardous is Part B to those partaking in the study?

"We rate the safety of Part B as a 3 due to its status in Phase 3, meaning that efficacy has been partially established and multiple safety assessments have occurred."

Answered by AI

What is the recruitment size of this clinical trial?

"That's right. According to the information on clinicaltrials.gov, recruitment is currently underway for this medical trial which was posted online June 27th 2023 and updated July 14th of the same year. A total of 64 patients are needed at two distinct sites."

Answered by AI

Is there an eligibility criteria for participants in this trial?

"64 cystic fibrosis patients aged between 12 and 24 months are being enrolled in this trial. Candidates must have at least one F508del mutation or another ELX/TEZ/IVA-responsive CFTR gene variant to qualify for inclusion."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

2023. "Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05882357.

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