Ivacaftor

Generic Name: Ivacaftor

Brand Names: Kalydeco, Orkambi, Symdeko, Trikafta (100 Mg / 50 Mg / 75 Mg; 150 Mg)

Drug Type: Small Molecule

Chemical Formula: C_₂₄H_₂₈N_₂O_₃

CAS Number: 873054-44-5

Unique Ingredient Identifier: 1Y740ILL1Z

Background: Ivacaftor (also known as Kalydeco or VX-770) is a drug used for the management of Cystic Fibrosis (CF). It is manufactured and distributed by Vertex Pharmaceuticals. It was approved by the Food and Drug Administration on January 31, 2012, and by Health Canada in late 2012. Ivacaftor is administered as a monotherapy and also administered in combination with o...

Indication: When used as monotherapy as the product Kalydeco, ivacaftor is indicated for the treatment of cystic fibrosis (CF) in patients aged one month and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
...

Associated Conditions: Cystic Fibrosis (CF)

Associated Therapies: -

Clinical Trials

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele

Phase 2

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: ELX-02
Drug: Ivacaftor

First Posted Date: 2019-10-22

Last Posted Date: 2022-11-22

Lead Sponsor: Eloxx Pharmaceuticals, Inc.

Target Recruit Count: 17

Registration Number: NCT04135495

Locations: 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
Stanford School of Medicine, Palo Alto, California, United States
🇺🇸
National Jewish Health, Denver, Colorado, United States

and more 7 locations

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

Phase 2

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: ELX-02
Drug: Ivacaftor

First Posted Date: 2019-10-15

Last Posted Date: 2023-08-21

Lead Sponsor: Eloxx Pharmaceuticals, Inc.

Target Recruit Count: 17

Registration Number: NCT04126473

Locations: 🇮🇱
Hadassah Medical Center, Jerusalem, Israel
🇩🇪
Universitätsklinikum Frankfurt, Frankfurt, Germany
🇦🇺
The Royal Prince Alfred Hospital, Camperdown, New South Whales, Australia

and more 6 locations

The Multicenter Topic Trial

Phase 2

Withdrawn

Conditions: Chronic Obstructive Pulmonary Disease

Interventions: Drug: Ivacaftor

First Posted Date: 2019-08-26

Last Posted Date: 2022-01-27

Lead Sponsor: University of Alabama at Birmingham

Registration Number: NCT04066751

Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects

Completed

Conditions: Cystic Fibrosis

First Posted Date: 2018-12-21

Last Posted Date: 2022-03-11

Lead Sponsor: Children's Hospital of Philadelphia

Target Recruit Count: 15

Registration Number: NCT03783286

Locations: 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Early Phase 1

Recruiting

Conditions: Chronic Rhinosinusitis (Diagnosis)

Interventions: Drug: Ivacaftor
Drug: standard of care treatment

First Posted Date: 2018-02-20

Last Posted Date: 2024-04-24

Lead Sponsor: University of Alabama at Birmingham

Target Recruit Count: 20

Registration Number: NCT03439865

Locations: 🇺🇸
University of Alabama at Birmingham, Birmingham, Alabama, United States

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Phase 3

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: Tezacaftor/Ivacaftor
Drug: Placebo
Drug: Ivacaftor

First Posted Date: 2017-05-12

Last Posted Date: 2019-09-12

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Target Recruit Count: 98

Registration Number: NCT03150719

Locations: 🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
Duke University Medical Center, Durham, North Carolina, United States

and more 50 locations

A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

Phase 3

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: Ivacaftor
Drug: Placebo

First Posted Date: 2017-03-01

Last Posted Date: 2020-02-26

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Target Recruit Count: 38

Registration Number: NCT03068312

Locations: 🇮🇱
Hadassah Medical Organization, Jerusalem, Israel

A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis

Phase 1

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: VX-659
Drug: VX-659 Matching Placebo
Drug: Tezacaftor
Drug: Ivacaftor
Drug: Triple Combination (TC) Matching Placebos

First Posted Date: 2017-01-24

Last Posted Date: 2017-09-05

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Target Recruit Count: 163

Registration Number: NCT03029455

An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

Phase 3

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: Ivacaftor

First Posted Date: 2016-10-17

Last Posted Date: 2018-08-06

Lead Sponsor: Medical University of South Carolina

Target Recruit Count: 2

Registration Number: NCT02934698

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

Phase 2

Completed

Conditions: Primary Ciliary Dyskinesia

Interventions: Drug: Hypertonic Saline
Drug: VX-371
Drug: VX-371 + HS
Drug: Placebo (0.17% saline)
Drug: Ivacaftor

First Posted Date: 2016-08-18

Last Posted Date: 2021-12-16

Lead Sponsor: Parion Sciences

Target Recruit Count: 123

Registration Number: NCT02871778

Drug Development Updates

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