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CFTR Modulator

Trikafta-based Therapies for Cystic Fibrosis

Phase 4

Recruiting

Research Sponsored by University of Alabama at Birmingham

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Body weight ≥ 16 kg

Age ≥ 18 yrs

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks

Awards & highlights

Study Summary

This trialwill test the effectiveness of two different treatments for a rare genetic disorder: one with two drugs, and one with one drug.

Who is the study for?

Adults with Cystic Fibrosis and a specific gene mutation (W1282X) can join this trial. They must be able to perform certain lung function tests, not have had recent changes in CF treatments or infections, and agree to use contraception if sexually active. Those with severe other conditions, recent major lung complications, or a history of substance abuse cannot participate.Check my eligibility

What is being tested?

The study is testing the effectiveness of combining different drugs (TEZ/IVA and Trikafta) compared to using ivacaftor alone for treating Cystic Fibrosis in patients with the W1282X mutation. The goal is to see if these combinations work better for improving lung function.See study design

What are the potential side effects?

Possible side effects from TEZ/IVA and Trikafta may include chest discomfort, trouble breathing, skin rashes or itching, stomach issues like pain or discomfort, liver problems indicated by blood tests, headache, dizziness, vision changes or cataracts.

Eligibility Criteria

Inclusion Criteria

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

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My body weight is at least 16 kg.

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I am 18 years old or older.

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My lung function, measured by FEV1, is between 30% and 90% of the expected value for my age, gender, and height.

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I have cystic fibrosis with a confirmed nonsense mutation in the CFTR gene.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

lung function

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

3Treatment groups

Experimental Treatment

Group I: TrikaftaExperimental Treatment1 Intervention

If the participant is not on a current modulator, they will take Trikafta for 28 days followed by a 28 day off period. This cycle will be continued for 168 days

Group II: Symdeko/TrikaftaExperimental Treatment1 Intervention

If the participant currently takes Symdeko , they will take Trikafta for a 28 day period followed by Symdeko for a 28 day period. This cycle will be continued for 168 days

Group III: Ivacaftor/TrikaftaExperimental Treatment1 Intervention

If the participant currently takes Ivacaftor , they will take Trikafta for a 28 day period followed by Ivacaftor for a 28 day period. This cycle will be continued for 168 days

0 / 4Eligibility criteria met