Ensuring Access to Optimal Therapy in CF: The ENACT Study

Официальное название

Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study

Обзор

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).

Подробное описание

Understanding variation in genetic response to pharmacological treatments and personalized CFTR modulator response is crucial to the optimization of the use of these novel compounds; expansion to all patients who might benefit from them; and development of predictive biomarkers. In addition, 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 and the downstream effects is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).

Вмешательства

• Препарат Elexacaftor / Ivacaftor / Tezacaftor
  This 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.
• Другое therapeutic drug monitoring
  Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.

Первичные конечные точки

• Concentration (ng/mL) [Срок оценки: 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.]

Вторичные конечные точки (3)

• Participant Mental and Neuropsychological Health [Срок оценки: From enrollment to the end of treatment at 6-12 months.]
• Investigators will evaluate the feasibility of reducing dose to manage Neuropsychological Side Effects (NPSE). [Срок оценки: From enrollment to study conclusion at 6-12 months (after all visits are completed).]
• Response to dosing adjustments [Срок оценки: From enrollment to study conclusion at 6-12 months, after all visits are completed.]

Критерии участия

Критерии включения

• 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
• clinically stable lung disease, defined as no documented acute decrease in FEV1 > 10%, OR use of additional antibiotics (intravenous \[IV\] or oral \[PO\]) within 4 weeks prior to screening

Критерии исключения

• 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
• in the opinion of the Investigator, medical or psychiatric illness, or other conditions that would interfere with participation

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 3 центра

• The University of Alabama at Birmingham — Birmingham
• Arkansas Children's Hospital — Little Rock
• University of Washington — Seattle

Идентификаторы

NCT: NCT07148739 · ENACT-NP · 1R01HL171034-01A1

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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