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

NCT04038710 | Completed

• 1 other identifier: HS-3235
• Study Type: observational
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• pulmonary function

  FEV1 values.

  For a year post initiation of therapy.

Secondary Outcomes (1)

• CFQ-R score

  For a year following the initiation of triple combination therapy.

Study Arms (1)

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

Triple combination therapy DRUG

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

Patients with severe disease

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Jewish Health: lead

Study Sites (1)

National Jewish Health

Denver, Colorado, 80206, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Derek Low, MD

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2019
• First Posted: July 31, 2019
• Study Start: September 5, 2019
• Primary Completion: February 26, 2021
• Study Completion: February 26, 2021
• Last Updated: March 11, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)