Trikafta in Cystic Fibrosis Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 8 April 2024
• Main ID: NCT03506061
• Date of registration: 13/04/2018
• Prospective Registration: Yes
• Primary sponsor: Emory University
• Public title: Trikafta in Cystic Fibrosis Patients
• Scientific title: iPS Cell Response to CFTR Modulators: Study of Trikafta in CF Patients Carrying Partial Function Mutations or N1303K CFTR
• Date of first enrolment: September 4, 2019
• Target sample size: 42
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03506061
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Eric Sorscher, MD

• Name: Eric Sorscher, MD
• Affiliation: Emory University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Provision of signed and dated informed consent form or assent form

- Stated willingness to comply with all study procedures and availability for the
duration of the study

- Male or female age =12

- A clinical diagnosis of CF or CFTR-related disease and either: 1) evidence for a
partial function mutation not currently covered or likely to be covered for treatment
with a CFTR modulator (Substudy 1), or 2) N1303K CFTR and a minimal function mutation
(Substudy 2)

- Sweat Chloride < 80 mmol/L and/or pancreatic sufficiency (no exogenous pancreatic
enzyme supplement therapy) or carrying the N1303K CFTR variant

- Able to perform spirometry meeting American Thoracic Society (ATS) criteria for
acceptability and repeatability

- Clinically stable in the past 4 weeks with no evidence of CF exacerbation (prior to
screening and study Day 1)

- Willingness to use at least one form of acceptable birth control including abstinence
or condom with spermicide. This will include birth control for at least one month
prior to screening and agreement to use such a method during study participation for
an additional four weeks after the last administration of study drug

- Ability to take Trikafta

- Agreement to adhere to all current medical therapies as designated by the CF care
center physician

Exclusion Criteria:

- Documented history of drug or alcohol abuse within the last year

- Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary
disease in the 4 weeks prior to screening

- Listed for lung or liver transplant at the time of screening

- Cirrhosis or elevated liver transaminases > 3 times the upper limit of normal

- Pregnant or breastfeeding

- Inhibitors or inducers of CYP3A4, including certain herbal medications and
grapefruit/grapefruit juice, or other medicines known to negatively influence Trikafta
administration

- History of solid organ transplant

- Active therapy for non-tuberculosis mycobacterial infection or any plan to initiate
non-tuberculosis mycobacterial therapies during the study period

- Known allergy to Trikafta

- Treatment in the last 6 months with an approved CFTR modulator

- Any other condition that in the opinion of the lead investigators might confound
results of the study or pose an additional risk from administering study drug

- Treatment with another investigational drug or other intervention within one month
prior to enrollment, throughout the duration of study participation, and for an
additional four weeks following final drug administration

- Evidence of cataract/lens opacity determined to be clinically significant by an
ophthalmologist at or within 3 months prior to the Screening Visit



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Trikafta

Primary Outcome(s)

Change in sweat chloride [Time Frame: Baseline, Days 14, 28, 56]

Change in forced expiratory volume in one second (FEV1) [Time Frame: Baseline, Days 7, 14, 28, 56]

Response of iPS cells to treatment [Time Frame: Baseline]

Secondary Outcome(s)

Change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Score [Time Frame: Baseline, Days 14, 28, 56]

Change in weight [Time Frame: Baseline, Days 14, 28, 56]

Secondary ID(s)

300001205

R01HL139876

IRB00108656

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation

National Heart, Lung, and Blood Institute (NHLBI)