CLINICAL TRIALS PROFILE FOR ELEXACAFTOR

Clinical Trials for Elexacaftor

Trial ID	Title	Status	Sponsor	Phase	Summary
NCT03506061 ↗	Trikafta in Cystic Fibrosis Patients	Recruiting	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	Cystic Fibrosis (CF) is a life threatening genetic disorder resulting from mutations found in the gene known as the cystic fibrosis transmembrane conductance regulator (CFTR). This clinical study will enroll 22 participants without the F508del mutation, carrying partial function 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), sweat chloride, and nasal potential difference. Additionally, the researchers will obtain skin biopsy material 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. It is hypothesized that a robust correlation will be established between in vitro Trikafta responsiveness of iPS cells and in vivo benefit (FEV1) to patients, and will provide a new tool for utilizing iPS to identify patient populations most suitable for cystic fibrosis modulator therapy.
NCT03506061 ↗	Trikafta in Cystic Fibrosis Patients	Recruiting	Emory University	Phase 2	Cystic Fibrosis (CF) is a life threatening genetic disorder resulting from mutations found in the gene known as the cystic fibrosis transmembrane conductance regulator (CFTR). This clinical study will enroll 22 participants without the F508del mutation, carrying partial function 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), sweat chloride, and nasal potential difference. Additionally, the researchers will obtain skin biopsy material 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. It is hypothesized that a robust correlation will be established between in vitro Trikafta responsiveness of iPS cells and in vivo benefit (FEV1) to patients, and will provide a new tool for utilizing iPS to identify patient populations most suitable for cystic fibrosis modulator therapy.
NCT03691779 ↗	Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age	Completed	Vertex Pharmaceuticals Incorporated	Phase 3	This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects 6 through 11 years of age with F/F and F/MF genotypes.
NCT04043806 ↗	A Study Evaluating the Long-term Safety of VX-445 Combination Therapy	Active, not recruiting	Vertex Pharmaceuticals Incorporated	Phase 3	This study will evaluate the long-term safety and tolerability of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA).

Clinical Trial Conditions for Elexacaftor

Condition Name

Condition Name
Intervention	Trials
Cystic Fibrosis	25
Cystic Fibrosis (CF)	1
Cystic Fibrosis Gastrointestinal Disease	1
Cystic Fibrosis in Children	1

Condition MeSH

Condition MeSH
Intervention	Trials
Cystic Fibrosis	26
Fibrosis	24
Gastrointestinal Diseases	1
Digestive System Diseases	1

Clinical Trial Locations for Elexacaftor

Trials by Country

Trials by Country
Location	Trials
United States	122
Australia	13
United Kingdom	12
Germany	9
Canada	9

Trials by US State

Trials by US State
Location	Trials
Washington	7
North Carolina	7
Colorado	7
California	7
Oregon	6

Clinical Trial Progress for Elexacaftor

Clinical Trial Phase

Clinical Trial Phase
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE2	1

Clinical Trial Status

Clinical Trial Status
Clinical Trial Phase	Trials
Recruiting	9
Not yet recruiting	8
Active, not recruiting	6

Clinical Trial Sponsors for Elexacaftor

Sponsor Name

Sponsor Name
Sponsor	Trials
Vertex Pharmaceuticals Incorporated	20
National Heart, Lung, and Blood Institute (NHLBI)	2
Emory University	2

Sponsor Type

Sponsor Type
Sponsor	Trials
Industry	22
Other	17
NIH	3