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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

Must not have

Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 2 weeks prior to screening

History of solid organ or hematological transplantation; positive hepatitis B surface antigen test; hepatitis C antibody test; or human immunodecifiency

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks

Awards & highlights

No Placebo-Only Group

Drug Has Already Been Approved

Pivotal Trial

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had a lung infection or cold in the last 2 weeks.

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I have had an organ transplant or tested positive for hepatitis B, C, or HIV.

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I currently smoke or have smoked 10 or more pack-years.

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I haven't had severe lung problems like coughing up a lot of blood, collapsed lung, or fluid around my lungs in the last month.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

lung function

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Trikafta

2019

Completed Phase 2

~50

0 / 8Eligibility criteria met