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CFTR Modulator

Symdeko for Cystic Fibrosis

Phase < 1

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

Age ≥6 y.o.

Stable CF pulmonary regimen

Must not have

Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)

Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 32 weeks

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This trial will test whether unapproved drugs can improve the function of CFTR mutations in people with CF.

Who is the study for?

This trial is for individuals with Cystic Fibrosis, aged 6 or older, who have specific CFTR mutations that might respond to certain drugs not yet approved for their condition. They must be on a stable CF treatment and able to give consent. People can't join if they have severe liver or kidney disease, had an organ transplant, are currently in another drug study, smoked recently, or take medications that don't mix well with the trial drugs.

What is being tested?

The trial is testing Symdeko in patients with Cystic Fibrosis who have certain genetic mutations. It's designed to see if this medication can help even though it's not officially approved for these particular mutations.

What are the potential side effects?

Symdeko may cause side effects such as headache, nausea, sinus congestion, dizziness and rash. More serious side effects could include elevated liver enzymes which would require monitoring by the healthcare team.

Eligibility Criteria

Inclusion Criteria

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

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I am 6 years old or older.

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My cystic fibrosis lung treatment has not changed recently.

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I have been diagnosed with cystic fibrosis.

Exclusion Criteria

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

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I cannot use CFTR modulators due to conditions like advanced cirrhosis or ESRD.

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I am not taking any medications that interact badly with others.

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I have had a solid organ transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 32 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~32 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 32 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

spirometry

Awards & Highlights

No Placebo-Only Group

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

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

3Treatment groups

Experimental Treatment

Group I: SymdekoExperimental Treatment1 Intervention

Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern

Group II: OrkambiExperimental Treatment1 Intervention

Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern

Group III: IvacaftorExperimental Treatment1 Intervention

Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.

0 / 6Eligibility criteria met