Status:

COMPLETED

: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)

Lead Sponsor:

National Jewish Health

Conditions:

Cystic Fibrosis (CF)

Eligibility:

All Genders

12+ years

Brief Summary

This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baselin...

Detailed Description

While cystic fibrosis (CF) therapeutic development previously targeted the signs and symptoms of the disease, in the last 5 years, two drugs that treat the basic defect in CF have been approved, ivaca...

Eligibility Criteria

Inclusion

  • Confirmed diagnosis of CF
  • Male or female subjects greater than or equal to 12 years of age
  • Ability to reproducibly perform spirometry testing
  • Physician decision to treat with tezacaftor/ivacaftor (Smydeko)
  • Ability to understand and sign a written informed consent or assent and comply with the requirements of the study
  • Continuous use of orkambi for at least 1 month prior to visit 1

Exclusion

  • History of hypersensitivity to tezacaftor and/or ivacaftor
  • Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data
  • Any acute lower respiratory symptoms treated with oral, inhaled or intravenous antibiotics (IV) or systemic corticosteroids within the 2 weeks prior to Visit 1
  • Major or traumatic surgery within 12 weeks prior to Visit 1
  • For women of child-bearing potential: a positive pregnancy test at Visit 1
  • Unable or unwilling to fast (including no enteric tube feedings) for at least 6 hours prior each visit
  • Initiation of any new chronic therapy within 4 weeks prior to Visit 1
  • Use of an investigational agent within 28 days prior to Visit 1
  • Use of chronic oral corticosteroids within 28 days prior to Visit 1
  • Treatment for nontuberculous mycobacterial (NTM) infection, consisting of greater than or equal to two antibiotics (oral, IV, and/or inhaled) within 28 days prior to Visit 1
  • History of lung or liver transplantation, or listing for organ transplantation

Key Trial Info

Start Date :

March 1 2018

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

November 1 2019

Estimated Enrollment :

5 Patients enrolled

Trial Details

Trial ID

NCT03445793

Start Date

March 1 2018

End Date

November 1 2019

Last Update

January 23 2024

Active Locations (1)

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1

National Jewish Heatlh

Denver, Colorado, United States, 80206