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CompletedNCT04038710Updated Mar 11, 2021

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

An observational study in Cystic Fibrosis, sponsored by National Jewish Health. Completed at 1 site in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2021-03-11.

Sponsored by National Jewish Health · Observational

Study type
Observational
Model
Case-only
Time perspective
Prospective
Enrollment
8
Ages
12 Years and older
Sex
All
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Study 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.

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Conditions studied

  • Cystic Fibrosis
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In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 8 is below the median of 85 across 482 observational studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

National Jewish Health is the lead sponsor of 108 studies on the registry; 14 are open to participants now.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
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.

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

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.
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Study design

Observational model
Case-only
Time perspective
Prospective
Enrollment
8 participants (actual)
Patient registry
No

Groups and cohorts

  • 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

Interventions

  • DrugTriple combination therapy

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

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What researchers measure

Primary outcomes

  1. pulmonary function

    FEV1 values.

    Time frame: For a year post initiation of therapy.

Secondary outcomes

  1. CFQ-R score

    cystic fibrosis questionnaire - revised has sections to measure pulmonary health and gastrointestinal health.

    Time frame: For a year following the initiation of triple combination therapy.

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Study locations

1 site
  • National Jewish Health
    Denver, Colorado 80206, United States
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 11, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04038710
Lead sponsor
National Jewish Health
Responsible party
Sponsor
First posted
Jul 31, 2019
Start date
Sep 5, 2019
Primary completion
Feb 26, 2021
Completion
Feb 26, 2021
Last update
Mar 11, 2021

Study contacts

Derek Low, MD
principal investigator · National Jewish Health

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Mar 2021. You cannot join it, but the record below documents what was studied.

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