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

NCT03445793 | Completed Results

• 1 other identifier: HS-3145
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 1

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 baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. Change from baseline at 6 months pre-specified will be reported. The length of study participation will be approximately 6 months.

Conditions

Cystic Fibrosis (CF)

Keywords

DeltaF508deletion (F508del) mutation in 2 copies

Outcome Measures

Primary Outcomes (1)

• Change in Sweat Chloride Concentration in Millimoles/Liter From Baseline at 6 Months Pre-specified to be Reported

  Sweat chloride is a measure of cystic fibrosis transmembrane conductance regulator function. The calculations represent the average change from baseline to the average change at 6 months.

  Baseline to 6 months

Secondary Outcomes (9)

• Rationale for Transition Per Physician Questionnaire

  1 day (the questionnaire is done once at visit 1)

• Rationale for Transition Per Subject Questionnaire

  1 day (the questionnaire is done once at visit 1)

• Pulmonary Exacerbations

  One year prior to study entry (time of consent) and during study participation

• Change in Percent Predicted (ppFEV1) Value From Baseline at 6 Months Pre-specified to be Reported

  Baseline to 6 months

• Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at 6 Months

  Baseline to 6 months

Eligibility Criteria

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

Study Population

Cystic fibrosis with 2 copies of F508del mutation.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• National Jewish Health: lead

Study Sites (1)

National Jewish Heatlh

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

Limitations and Caveats

The limitation is the small sample size. Less patients with cystic fibrosis transitioned from lumacaftor/ivacaftor to tezacaftor/ivacaftor than expected. The enrolled number of patients left the study underpowered to detect differences.

Results Point of Contact

• Title: Jennifer Taylor-Cousar, Principal Investigator
• Organization: National Jewish Health

taylorcousarj@njhealth.org

3032702764

Study Officials

• Jennifer Taylor-Cousar, MD, MSCS

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Principal Investigator, Co-Director/CF TDN Director, Adult CF Program

Study Record Dates

• First Submitted: February 5, 2018
• First Posted: February 26, 2018
• Study Start: March 1, 2018
• Primary Completion: November 1, 2019
• Study Completion: November 1, 2019
• Last Updated: January 23, 2024
• Results First Posted: October 1, 2021

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)