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Recruiting NCT03587961

Personalized Theratyping Trial

Early Phase I Interventional Cystic Fibrosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Symdeko.
Who it may be relevant to
Registry conditions: Cystic Fibrosis. Basic parameters: from 6 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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.

Detailed description

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. Symdeko (Tezacaftor/Ivacaftor), Orkambi (Ivacaftor and Lumacaftor), correctors of CFTR misfolding and Kalydeco (Ivacaftor), a potentiator of abnormal CFTR gating, will be explored as a treatment for patients with other CF mutations than those currently approved. Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, or Orkambi, depending on the in vitro response pattern. 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. If the patient is 6-12 years old, we will only study Orkambi or ivacaftor as symdeko is not yet FDA approved in this patient population.

Interventions

  • Drug Symdeko
    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.

Primary outcome measures

  • spirometry [Time frame: 32 weeks]

Eligibility criteria

Inclusion criteria

  • Diagnosis of CF
  • Age ≥6 y.o.
  • CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators
  • Informed Consent/Assent
  • Stable CF pulmonary regimen

Exclusion criteria

  • Exacerbation requiring antibiotic or steroids for >28 days before trial entry
  • Ongoing participation in a CFTR modulator study
  • Active smoking in the past 6 months
  • History of solid organ transplant
  • Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)
  • Any condition that precludes the patient from participation in the opinion of the investigator
  • Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • University of Alabama at Birmingham — Birmingham

Identifiers

NCT: NCT03587961 · IRB-300001867 · IMPACT-Solomon

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗