20 Participants Needed

Symdeko for Cystic Fibrosis

HH
Overseen ByHeather Hathorne, PhD
Age: Any Age
Sex: Any
Trial Phase: Phase < 1
Sponsor: University of Alabama at Birmingham
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it excludes those taking medications with significant drug interactions with CFTR modulators. It's best to discuss your specific medications with the study team.

What data supports the effectiveness of the drug Symdeko for treating cystic fibrosis?

Research shows that the combination of tezacaftor and ivacaftor, which is part of Symdeko, is effective in improving lung function and quality of life in people with cystic fibrosis. It has a favorable safety profile and is particularly beneficial for those with specific genetic mutations related to cystic fibrosis.12345

Is Symdeko (tezacaftor/ivacaftor) safe for people with cystic fibrosis?

Symdeko (tezacaftor/ivacaftor) has been studied for safety in people with cystic fibrosis, especially those who had to stop another treatment due to breathing problems. It generally has a good safety profile, but some people may experience side effects like rash or headache, and liver function should be monitored.45678

How is the drug Symdeko different from other cystic fibrosis treatments?

Symdeko, which combines tezacaftor and ivacaftor, is unique because it targets the underlying cause of cystic fibrosis by helping the defective protein function better, specifically for those with certain genetic mutations. This approach is different from treatments that only manage symptoms, offering a more targeted therapy for improving lung function and overall health in eligible patients.5791011

What is the purpose of this trial?

The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

Eligibility Criteria

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.

Inclusion Criteria

I am 6 years old or older.
My CFTR mutation could improve with specific approved treatments.
Informed Consent/Assent
See 2 more

Exclusion Criteria

I cannot use CFTR modulators due to conditions like advanced cirrhosis or ESRD.
I am not taking any medications that interact badly with others.
You have smoked cigarettes within the last 6 months.
See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive off-label CFTR modulators such as Symdeko, Orkambi, or Ivacaftor based on their CFTR mutation response

32 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Symdeko
Trial Overview 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.
Participant Groups
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.

Symdeko is already approved in United States, Canada, European Union for the following indications:

🇺🇸
Approved in United States as Symdeko for:
  • Cystic fibrosis in people ages 12 and older with certain mutations in the CFTR gene
🇨🇦
Approved in Canada as Symdeko for:
  • Cystic fibrosis in people with certain mutations in the CFTR gene
🇪🇺
Approved in European Union as Symkevi for:
  • Cystic fibrosis in people with certain mutations in the CFTR gene

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Alabama at Birmingham

Lead Sponsor

Trials
1,677
Recruited
2,458,000+

Findings from Research

In a study of 48 adults with cystic fibrosis, treatment with tezacaftor-ivacaftor (TEZ/IVA) led to significant improvements in lung function (FEV1 increased from 66% to 72%) and reduced annual acute exacerbation rates after 6 months.
TEZ/IVA was found to be safe and well-tolerated, with only 6% of participants experiencing adverse events, indicating its potential as an effective treatment option for adults with cystic fibrosis.
Real-world data on the efficacy and safety of tezacaftor-ivacaftor in adults living with cystic fibrosis homozygous for F508del and heterozygous for F508del and a residual function mutation.Vincken, S., Verbanck, S., Braun, S., et al.[2023]
In a phase 3b trial involving 176 participants with cystic fibrosis homozygous for the F508del-CFTR mutation, the triple-combination therapy of elexacaftor, tezacaftor, and ivacaftor resulted in significant improvements in respiratory quality of life and lung function over 24 weeks, outperforming the dual therapy of tezacaftor and ivacaftor.
The treatment was found to be safe and well-tolerated, with most adverse events being mild or moderate, and only a small percentage of participants experiencing serious adverse events.
Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial.Sutharsan, S., McKone, EF., Downey, DG., et al.[2022]
CFTR modulators, like tezacaftor/ivacaftor, represent a significant advancement in treating cystic fibrosis by targeting the underlying genetic defect, with promising results from pivotal clinical trials.
Tezacaftor/ivacaftor has a better safety profile compared to lumacaftor/ivacaftor and is effective for a wider range of CFTR mutations, paving the way for future therapies that could benefit most cystic fibrosis patients.
Tezacaftor and ivacaftor for the treatment of cystic fibrosis.Paterson, SL., Barry, PJ., Horsley, AR.[2021]

References

Real-world data on the efficacy and safety of tezacaftor-ivacaftor in adults living with cystic fibrosis homozygous for F508del and heterozygous for F508del and a residual function mutation. [2023]
Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial. [2022]
Tezacaftor and ivacaftor for the treatment of cystic fibrosis. [2021]
Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy. [2020]
Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis. [2023]
Tezacaftor/ivacaftor in people with cystic fibrosis who stopped lumacaftor/ivacaftor due to respiratory adverse events. [2022]
Phase 1 Study to Assess the Safety and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Subjects Without Cystic Fibrosis With Moderate Hepatic Impairment. [2022]
If At First You Don't Succeed, Trikafta Again. [2022]
Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease. [2022]
Elexacaftor/Tezacaftor/Ivacaftor as a Bridge to Lung Retransplant in a Recipient With Cystic Fibrosis. [2022]
Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations. [2021]
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