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OpenTrials
Completed

NCT Number: NCT04038710

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

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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Key information

Conditions

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

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

National Jewish Health

Denver, Colorado, 80206, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Triple combination therapy

Drug

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

Primary outcomes

  1. pulmonary function

    Time frame: For a year post initiation of therapy.

    FEV1 values.

Secondary outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

National Jewish Health

Other

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Jul 31, 2019
Registry last updated
Mar 11, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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