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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04038710
• Other study ID #: HS-3235
• Status: Completed
• Start date: September 5, 2019
• Est. completion date: February 26, 2021

Study information

• Verified date: March 2021
• Source: National Jewish Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: February 26, 2021
• Est. primary completion date: February 26, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado

Sponsors (1)

Lead Sponsor	Collaborator
National Jewish Health

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	pulmonary function	FEV1 values.	For a year post initiation of therapy.
Secondary	CFQ-R score	cystic fibrosis questionnaire - revised has sections to measure pulmonary health and gastrointestinal health.	For a year following the initiation of triple combination therapy.