Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.

An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04038710

Enrollment

Registered

2019-07-31

Start date

2019-09-05

Completion date

2021-02-26

Last updated

2021-03-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Brief summary

This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these people with severe cystic fibrosis disease.

Interventions

DRUGTriple combination therapy

Triple combination is compromised of the drugs ivacaftor, tezacaftor and Vertex's next generation modulator.

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

12 Years to No maximum

Healthy volunteers

Inclusion criteria

* Confirmed diagnosis of Cystic Fibrosis * Ability to reproducibly perform spirometry (according to ATS criteria) * Physician decision to treat with TCT through the EAP program * Ability to understand and sign a written informed consent or assent and comply with the requirements of the study

Exclusion criteria

* History of hypersensitivity to VX 445, VX 659, tezacaftor and/or ivacaftor * Any acute lower respiratory symptoms treated with oral, inhaled or IV antibiotics or systemic corticosteroids within the 2 weeks prior to Visit 1. * Major or traumatic surgery within 12 weeks prior to Visit 1. * Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1. * Use of an investigational agent within 28 days prior to Visit 1. * History of lung or liver transplantation or listing for organ transplantation.

Design outcomes

Primary

Measure	Time frame	Description
pulmonary function	For a year post initiation of therapy.	FEV1 values.

Secondary

Measure	Time frame	Description
CFQ-R score	For a year following the initiation of triple combination therapy.	cystic fibrosis questionnaire - revised has sections to measure pulmonary health and gastrointestinal health.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026