Ensuring Access to Optimal Therapy in CF: The ENACT Study
ConditionCystic Fibrosis (CF)
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age3+
SponsorArkansas Children's Hospital Research Institute
About this trial
This clinical trial is examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic factors are examined. The hope is that the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
documentation of CF diagnosis per CFF diagnostic criteria and known CFTR genotype
age 2 years and older
ability to provide written informed consent and/or assent (by subject and/or legal guardian)
on a stable dose of triple combination CFTR modulator therapy for at least two weeks prior to Visit 1
Disqualifiers
recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening
pregnant or breastfeeding female
history of alcohol or substance abuse in the 6 months prior to screening
participation in a study involving an investigational intervention within 28 days (or 5 half-lives, whichever is longer) prior to screening
Trial design
Design model
Single group
Treatments tested in this trial
Elexacaftor / Ivacaftor / Tezacaftor
DrugThis study will examine different dosing strategies and outcomes for triple combination CFTR modulator therapy using the drug(s) elexacaftor, tezacaftor, and/or ivacaftor in patients with cystic fibrosis.
therapeutic drug monitoring
Other interventionParticipants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.
Treatment groups
100 Participants
are divided into 1 treatment groupGroup A: Single ArmOther 2 interventions
Trial outcomes
Primary outcomes
1
Concentration (ng/mL)
Drug concentration of CFTR modulators
Time frame
One time assessment for observational part of the study, up to 6 times (6 months or more) for the therapeutic drug monitoring pilot and feasibility study.Secondary outcomes
1
Participant Mental and Neuropsychological Health
Participants will respond to questionnaires about anxiety, depression, suicidal ideation and other indicators of mental and neuropsychological health. Participants will complete assessments and an exam at each study visit.
Time frame
From enrollment to the end of treatment at 6-12 months.2
Investigators will evaluate the feasibility of reducing dose to manage Neuropsychological Side Effects (NPSE).
The primary goal will be to assess feasibility to monitor clinical outcomes, patient receptiveness, collection of samples, and concentration interpretation. Assessment of feasibility will include the patient acceptance of dose reduction using an acceptability questionnaire, turnaround for quantitation results by timely return of result, and clinical appropriateness for dose reduction (by monitoring clinical response).
Time frame
From enrollment to study conclusion at 6-12 months (after all visits are completed).3
Response to dosing adjustments
Outcome includes symptom assessment, spirometry, sweat chloride, weight, and TC quantitation at Visits 2-6. Investigators will monitor patients for 6 visits to assess CF and NP symptom stability under the supervision of the licensed clinical psychologist and TC concentration variability over time. At each visit investigators will re-assess dosing strategy.
Time frame
From enrollment to study conclusion at 6-12 months, after all visits are completed.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Arkansas Children's Hospital Research Institute
Lead sponsor
National Institutes of Health (NIH)
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Source ClinicalTrials.gov