Phase 2
Completed N=42
Trikafta in Cystic Fibrosis Patients
Source: ClinicalTrials.gov NCT03506061 ↗Enrolled (actual)
42
Serious AEs
2.4%
Results posted
Sep 2025
Primary outcomePrimary: Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function — 74.8; 76 percent of predicted FEV1
Summary
This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta, and who are not expected to be approved for CFTR modulator treatment in the immediate future. Each participant will be given Trikafta for approximately four weeks. The study researchers will monitor clinical endpoints that include forced expiratory volume (FEV1) and sweat chloride. Additionally, the researchers will obtain skin biopsy material and/or blood sample from each subject so that induced pluripotent stem (iPS) cells can be modified into airway cell monolayers and tested for response to Trikafta. In this way, the study will evaluate an emerging and readily accessible in vitro endpoint as a predictor of clinical response. This study will serve as a pilot/test case for other clinical protocols relevant to patients with rare CFTR variants who do not currently receive modulator therapies.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function |
74.8; 76 | — |
| PRIMARY Sweat Chloride Among Participants Who Encode the N1303K Variant |
109; 107.9 | — |
| PRIMARY Number of Participants With Induced Pluripotent Stem (iPS) Cells Predicting Response to Treatment Among Participants With Evidence of Partial Function |
— | — |
| SECONDARY Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants Who Encode the N1303K Variant |
75.8; 85.3 | — |
| SECONDARY Sweat Chloride Among Participants With Evidence of Partial Function |
66.9; 57.8 | — |
| SECONDARY Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score |
66.4; 60.6; 74.1; 81.4 | — |
| SECONDARY Weight |
75; 57.5; 75.8; 58.5 | — |
| SECONDARY Body Mass Index (BMI) |
25.4; 22.1; 25.7; 22.5 | — |
| SECONDARY Number of Participants With iPS Cells Predicting Response to Treatment Among Participants Encoding N1303K |
— | — |
Eligibility 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 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
Data sourced from ClinicalTrials.gov (NCT03506061). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.