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).
NCT04058353 ↗	A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)	Completed	Vertex Pharmaceuticals Incorporated	Phase 3	This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).
NCT04058366 ↗	Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy	Active, not recruiting	Vertex Pharmaceuticals Incorporated	Phase 3	This study will evaluate the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).

Clinical Trial Conditions for Elexacaftor

Condition Name

Condition Name for
Intervention	Trials
Cystic Fibrosis	23
Adherence, Medication	1
Cystic Fibrosis Gastrointestinal Disease	1
Cystic Fibrosis in Children	1

Condition MeSH

Condition MeSH for
Intervention	Trials
Fibrosis	24
Cystic Fibrosis	23
Bronchiectasis	1
Liver Diseases	1

Clinical Trial Locations for Elexacaftor

Trials by Country

Trials by Country for
Location	Trials
United States	119
Australia	12
United Kingdom	11
Germany	8
Canada	8

Trials by US State

Trials by US State for
Location	Trials
Colorado	7
California	7
North Carolina	7
Missouri	6
Minnesota	6

Clinical Trial Progress for Elexacaftor

Clinical Trial Phase

Clinical Trial Phase for
Clinical Trial Phase	Trials
Phase 4	4
Phase 3	19
Phase 2	1

Clinical Trial Status

Clinical Trial Status for
Clinical Trial Phase	Trials
Not yet recruiting	8
Recruiting	7
Active, not recruiting	6

Clinical Trial Sponsors for Elexacaftor

Sponsor Name

Sponsor Name for
Sponsor	Trials
Vertex Pharmaceuticals Incorporated	19
Emory University	2
Teagasc	1

Sponsor Type

Sponsor Type for
Sponsor	Trials
Industry	21
Other	15
NIH	1