70 Participants Needed

ELX/TEZ/IVA for Cystic Fibrosis

Recruiting at 18 trial locations
MI
Overseen ByMedical Information
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Vertex Pharmaceuticals Incorporated
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\<) 24 months of age.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinator or your doctor for guidance.

How is the drug ELX/TEZ/IVA unique for treating cystic fibrosis?

ELX/TEZ/IVA is unique because it combines three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor) to improve the function of the CFTR protein in cystic fibrosis patients, especially those with the F508del mutation. This combination has shown significant improvements in lung function and other health outcomes compared to previous treatments.12345

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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

Exclusion Criteria

History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

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

8 weeks

Treatment Part B

Participants receive ELX/TEZ/IVA in the morning and IVA in the evening with doses based on Part A outcomes

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • ELX/TEZ/IVA
  • IVA
Trial Overview 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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Part BExperimental Treatment2 Interventions
Group II: Part AExperimental Treatment2 Interventions

ELX/TEZ/IVA is already approved in United States, European Union, Canada for the following indications:

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Approved in United States as Trikafta for:
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Approved in European Union as Kaftrio for:
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Approved in Canada as Trikafta for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials
267
Recruited
36,100+
Dr. David Altshuler profile image

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani profile image

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

Published Research Related to This Trial

In a study of 13 lung transplant recipients (LTRs) using elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), the therapy was found to be poorly tolerated, with 38.4% of patients discontinuing due to issues like declining pulmonary function and mood disturbances.
Despite the challenges, 46.2% of patients reported improvements in sinus symptoms and a significant reduction in tacrolimus dose requirements (by 50%), indicating some therapeutic benefit for extrapulmonary cystic fibrosis manifestations.
Poor tolerability of cystic fibrosis transmembrane conductance regulator modulator therapy in lung transplant recipients.Doligalski, CT., McKinzie, CJ., Yang, A., et al.[2022]
In a study of 19 cystic fibrosis patients treated with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) for 6 months, significant improvements were observed in lung structure, including reduced bronchial wall thickening and mucus plugging, as assessed by MRI.
Patients also showed marked clinical benefits, including improved respiratory function (FEV1% increased from 58.5% to 71.4%), better body mass index, and reduced pulmonary exacerbations, confirming the efficacy of ELX/TEZ/IVA in managing cystic fibrosis.
Structural changes in lung morphology detected by MRI after modulating therapy with elexacaftor/tezacaftor/ivacaftor in adolescent and adult patients with cystic fibrosis.Fainardi, V., Skenderaj, K., Ciuni, A., et al.[2023]
In a study of 16,116 cystic fibrosis patients treated with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) for an average of 20.4 months, there was a significant 79% reduction in pulmonary exacerbations and a 74% reduction in hospitalizations compared to the 5-year pre-treatment period.
The treatment also led to sustained improvements in lung function (average increase of 8.9 percentage points in forced expiratory volume in 1 second by Year 2), increased body mass index, and a notable decrease in the rates of death (72% lower) and lung transplantation (85% lower) compared to the year before the treatment became available.
Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study.Bower, JK., Volkova, N., Ahluwalia, N., et al.[2023]

Citations

Poor tolerability of cystic fibrosis transmembrane conductance regulator modulator therapy in lung transplant recipients. [2022]
Structural changes in lung morphology detected by MRI after modulating therapy with elexacaftor/tezacaftor/ivacaftor in adolescent and adult patients with cystic fibrosis. [2023]
Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study. [2023]
Acneiform Eruption Following Elexacaftor-Tezacaftor-Ivacaftor Treatment in Patients With Cystic Fibrosis. [2023]
Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation. [2023]
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