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

March 9, 2021 updated by: National Jewish Health

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

8

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients at National Jewish Health with Cystic Fibrosis who are eligible to enroll in Vertex's triple combination therapy expanded access program.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with severe disease
Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program.
Drug: Triple combination therapy
Triple combination is compromised of the drugs ivacaftor, tezacaftor and Vertex's next generation modulator.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
pulmonary function
Time Frame: For a year post initiation of therapy.
FEV1 values.
For a year post initiation of therapy.

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Jewish Health

Investigators

  • Principal Investigator: Derek Low, MD, National Jewish Health

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 5, 2019

Primary Completion (Actual)

February 26, 2021

Study Completion (Actual)

February 26, 2021

Study Registration Dates

First Submitted

July 26, 2019

First Submitted That Met QC Criteria

July 26, 2019

First Posted (Actual)

July 31, 2019

Study Record Updates

Last Update Posted (Actual)

March 11, 2021

Last Update Submitted That Met QC Criteria

March 9, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HS-3235

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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