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

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: Triple combination therapy

• Registration Number: NCT04038710
• Lead Sponsor: National Jewish Health

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-07-26
• Study First Submitted QC: 2019-07-26
• Study First Posted: 2019-07-31
• Study Start: 2019-09-05
• Primary Completion: 2021-02-26
• Study Completion: 2021-02-26
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-09
• Last Update Posted: 2021-03-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with severe disease	Triple combination therapy	Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program.

Primary Outcome Measures

Name	Time	Method
pulmonary function	For a year post initiation of therapy.	FEV1 values.

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

National Jewish Health; 🇺🇸 Denver, Colorado, United States