Getting insight in the exposure effect relationship of Elexacaftor/ Tezacaftor/Ivacaftor treatment in people with cystic fibrosis
- Conditions
- Cystic fibrosisMucoviscoidosis1003868610010613
- Registration Number
- NL-OMON56478
- Lead Sponsor
- HagaZiekenhuis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 20
In order to be eligible to participate in this study, a subject must meet all
of the following criteria:
1.Cystic fibrosis based on DBC code ( specialisme: 0322 ; Diagnose: 1403)
2.Use of Kaftrio tablets with dosage of 75/5-/100mg and ATC code: R07AX32
3.Use of standard dose of ETI (2 tablets in the morning) and Ivacaftor (1
tablet in the evening).
4.Availability of a baseline and on ETI treatment sweat chloride value
5.A homozygous F508del mutation in the CFTR gene
6.A stable clinical condition defined as no pulmonary exacerbation in a period
of 4 weeks before study visit
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
1. Solid organ transplantation
2. The use of concomitant medications with CYP3A4 interactions during the
period of determining the trough concentration of ETI
3. Pregnancy
4. Liver cirrhosis
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The study will involve the determination of the trough concentration (Ctrough)<br /><br>of ETI in all 20 collected blood samples for statistical analysis. We will<br /><br>compare Ctrough of the group with the highest absolute sweat chloride decrease<br /><br>to C trough levels of the group with the lowest absolute sweat chloride<br /><br>decrease. Since our study group is small, we will use a non-parametric,<br /><br>Mann-whitney u test to compare the Ctrough of ETI in both groups. </p><br>
- Secondary Outcome Measures
Name Time Method <p>not applicable</p><br>