N/A
Completed N=5
: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
Cystic Fibrosis (CF)
Source: ClinicalTrials.gov NCT03445793 ↗
Enrolled (actual)
5
Serious AEs
60.0%
Results posted
Oct 2021
Primary outcomePrimary: Change in Sweat Chloride Concentration in Millimoles/Liter From Baseline at 6 Months Pre-specified to be Reported — 10.2 mmol/L
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Sweat Chloride Concentration in Millimoles/Liter From Baseline at 6 Months Pre-specified to be Reported |
10.2 | — |
| SECONDARY Rationale for Transition Per Physician Questionnaire |
4; 1 | — |
| SECONDARY Rationale for Transition Per Subject Questionnaire |
1; 1; 2; 1 | — |
| SECONDARY Pulmonary Exacerbations |
3.2 | — |
| SECONDARY Change in Percent Predicted (ppFEV1) Value From Baseline at 6 Months Pre-specified to be Reported |
-2.0 | — |
| SECONDARY Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at 6 Months |
-11.3 | — |
| SECONDARY Change in Weight in Kilograms From Baseline at 6 Months Pre-specified to be Reported |
2.0 | — |
| SECONDARY Change in BMI in kg/m^2 From Baseline at 6 Months Pre-specified to be Reported |
-0.67 | — |
| SECONDARY Number of People With Undetectable or Normal Fecal Elastase Measurements at 6 Months Pre-specified to be Reported |
5; 0 | — |
| SECONDARY Change in Gastrointestinal Symptom Tracker Score From Baseline to 6 Months |
0.6 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT03445793). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.