A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Subjects with Cystic Fibrosis (CF)

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2021-001628-16-ES

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 82

Inclusion Criteria

1.Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form.
2.Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
3.Subjects (male and female) 12 years of age or older on the date of informed consent.
4.Subjects heterozygous for F508del and an MF mutation (F/MF genotypes, see Protocol Appendix A. for definition and non-exhaustive list of eligible MF mutations).
a. Genotype should be confirmed at the Screening Visit.
b.If the screening CFTR genotype result is not received before the first dose of study drug, a previous CFTR genotype laboratory report may be used to establish eligibility.
c. Subjects who have been enrolled and whose screening genotype does not confirm study eligibility must be discontinued from the study (Protocol Section9.9).
5.Forced expiratory volume in 1 second (FEV1) value =30% and =90% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit (spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability) and stable CF disease as judged by the investigator.
6.Willing to remain on a stable CF treatment regimen (other than CFTR modulators) through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 75
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1.History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following:
•Clinically significant liver cirrhosis with or without portal hypertension
•Solid organ or hematological transplantation (or on a transplant list)
•Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator
•Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage0 cervical carcinoma in situ (all3 with no recurrence for the last 5years)
•Non-ambulatory status
2.Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (as deemed by the investigator).
3.Any of the following abnormal laboratory values at screening:
•Hemoglobin <10 g/dL
•Total bilirubin =2 × upper limit of normal (ULN)
•Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) =3×ULN
•Abnormal renal function defined as glomerular filtration rate =50mL/min/1.73m2(calculated by the Modification of Diet in Renal Disease Study Equation) for subjects =18years of age and =45mL/min/1.73m2(calculated by the Counahan-Barratt equation) for subjects <18 years of age.
4.An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
5.Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:
•The subject has not had a respiratory tract culture positive for these organisms within the 12months before the date of informed consent.
•The subject has had at least 2respiratory tract cultures negative for such organisms within the 12months before the date of informed consent, with the first and last of these separated by at least 3months, and the most recent one within the 6months before the date of informed consent.
6.An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug (Day 1).
7.Ongoing or prior participation in an investigational drug study within 28days of the Screening Visit.
•A washout period of 5terminal half-lives of the previous investigational study drug, or 28 days, whichever is longer, must elapse before the Screening Visit.
•The duration of the elapsed time may be longer if required by local regulations.
8.Use of restricted medication within specified duration before the first dose of study drug as defined in Protocol Table 9-1.
9.Pregnant and breast-feeding females. All female subjects regardless of childbearing potential (Protocol Section11.4.6) must have a negative pregnancy test at the Screening Visit and the Day1 Visit.
10.The subject or a close relative of the subject is the investigator or a sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adul

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effects of ELX/TEZ/IVA on cough and physical activity using wearable technology;Secondary Objective: Not applicable;Primary end point(s): Percent reduction from baseline in cough frequency (cough events per day) to the average of Week 8 through Week 12;Timepoint(s) of evaluation of this end point: From baseline to week 12

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Absolute change from baseline in total step count per day to the average of Week 8 through Week 12;Timepoint(s) of evaluation of this end point: From baseline to Week 12

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