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
Qualifiers
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
Treatments tested in this trial
- Elexacaftor / Ivacaftor / Tezacaftor
- therapeutic drug monitoring
Treatment groups
100 Participants
are divided into 1 treatment groupSponsors and collaborators
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