Cystic Fibrosis (CF) Clinical Trial
Official title:
: TRANSITION: An Observational Study of the Effects on Sweat Chloride and Clinical Outcomes of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
NCT number | NCT03445793 |
Other study ID # | HS-3145 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | March 1, 2018 |
Est. completion date | November 1, 2019 |
Verified date | January 2024 |
Source | National Jewish Health |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 5 |
Est. completion date | November 1, 2019 |
Est. primary completion date | November 1, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years and older |
Eligibility | Inclusion Criteria: - 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 Criteria: - 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 |
Country | Name | City | State |
---|---|---|---|
United States | National Jewish Heatlh | Denver | Colorado |
Lead Sponsor | Collaborator |
---|---|
National Jewish Health |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | Rationale for Transition Per Physician Questionnaire | Questionnaire to determine the treating physician's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor | 1 day (the questionnaire is done once at visit 1) | |
Secondary | Rationale for Transition Per Subject Questionnaire | Questionnaire to determine the subject's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor | 1 day (the questionnaire is done once at visit 1) | |
Secondary | Pulmonary Exacerbations | Number of pulmonary exacerbations requiring oral or IV antibiotics | One year prior to study entry (time of consent) and during study participation | |
Secondary | Change in Percent Predicted (ppFEV1) Value From Baseline at 6 Months Pre-specified to be Reported | Pulmonary function by spirometry, percent predicted forced expiratory volume in 1 second. The calculations represent the average change from baseline to the average change at 6 months. | Baseline to 6 months | |
Secondary | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at 6 Months | CF-related quality of life measure is a validated, CF specific, patient reported outcome (PRO). This portion of the PRO is specific to respiratory symptoms. The scaled score for each domain ranges from 0 (worst condition) to 100 (best condition), with higher scores indicating better health in respiratory domain. | Baseline to 6 months | |
Secondary | Change in Weight in Kilograms From Baseline at 6 Months Pre-specified to be Reported | Weight is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months. | Baseline to 6 months | |
Secondary | Change in BMI in kg/m^2 From Baseline at 6 Months Pre-specified to be Reported | BMI is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months. | Baseline to 6 months | |
Secondary | Number of People With Undetectable or Normal Fecal Elastase Measurements at 6 Months Pre-specified to be Reported | Fecal elastase in micrograms/gram. Fecal elastase is a measure of pancreatic function: Less than 100 mcg/g indicates severe exocrine pancreatic insufficiency, 100-200 mcg/g indicates moderate to mild insufficiency, greater than 200 indicates normal pancreatic function. The count represents the number of participants with either an undetectable fecal elastase level or normal level at 6 months, indicating change in pancreatic function at 6 months. | 6 months | |
Secondary | Change in Gastrointestinal Symptom Tracker Score From Baseline to 6 Months | The Exocrine Pancreatic Insufficiency (EPI) GI Symptom Tracker is meant to measure the impact treatment is having on GI symptoms, perceived by person completing the tracker. Participants answer 24 symptom questions on a scale from almost always to never (scale 4 to 1). Higher scores reflect more challenges/problems related to GI symptoms. For purposes of this outcome measure, subscales were combined to compute a total score (minimum score 24, maximum score 96). The difference of total scores from baseline at 6 months were calculated to measure average overall change in GI symptoms. | Baseline to 6 months |
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