NCT04038710CompletedNot Applicable
An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- National Jewish Health
- Enrollment
- 8
- Locations
- 1
- Primary Endpoint
- pulmonary function
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Case Only
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
- •Major or traumatic surgery within 12 weeks prior to Visit
- •Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit
- •Use of an investigational agent within 28 days prior to Visit
- •History of lung or liver transplantation or listing for organ transplantation.
Arms & Interventions
Patients with severe disease
Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program.
Intervention: Triple combination therapy (Drug)
Outcomes
Primary Outcomes
pulmonary function
Time Frame: For a year post initiation of therapy.
FEV1 values.
Secondary Outcomes
- CFQ-R score(For a year following the initiation of triple combination therapy.)
Investigators
Study Sites (1)
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