Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Through 11 Years of Age

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: EUCTR2021-005930-40-SE
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-09-29
• Last Update Posted: 9 January 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 65

Inclusion Criteria

Cohorts B1
1. Subject (or subject’s legally appointed and authorized representative) will sign and date an ICF, and an assent form.
2. Subjects 6 through 11 years of age, on the date of informed consent; subjects who completed Cohort A1 but are =12 years of age on the date of informed consent in Cohort B1 are not eligible to enroll in Cohort B1.
3. Subjects whose weight is between the 5th and 95th percentile for weight-for-age at the Screening Visit based on current CDC growth charts.
4. Confirmed diagnosis of CF as determined by the investigator.
5. Subjects who have at least 1 TCR mutation (including F508del) in the CFTR gene.
6. Subjects with FEV1 =60% of predicted normal for age, sex, and height using equations of the Global Lung Function Initiative at the Screening Visit.
7. Subjects with stable CF disease at the start of the Treatment Period as deemed by the investigator.
8. Subjects who are willing to remain on a stable CF medication regimen (other than CFTR modulators) through through Week 24 (Cohort B1) or, if applicable, through the Safety Follow-up Visit.
9. Subjects who are able to swallow tablets.
10. Female subjects of childbearing potential must have a negative serum pregnancy test at the Screening Visit.
11. Subjects of childbearing potential and who are sexually active must meet the contraception requirements.
Are the trial subjects under 18? yes
Number of subjects for this age range: 65
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Cohorts B1
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:
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
• Chronic kidney disease of Stage 3 or above
• Solid organ or hematological transplantation
• Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator
• Cancer
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.
3. History of intolerance to study drug that would pose an additional risk to the subject in the opinion of the investigator.
4. Any of the following abnormal laboratory values at screening:
• Hemoglobin <10 g/dL
• Total bilirubin =2 × ULN
• AST, ALT, GGT, or ALP =3 × ULN
• Abnormal renal function defined as glomerular filtration rate =45 mL/min/1.73 m2
5. An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 .
6. Lung infection with organisms associated with a more rapid decline in pulmonary status.
7. An acute illness not related to CF within 14 days before Day 1.
8. Ongoing or prior participation in a study of an investigational treatment other than a Vertex CFTR modulator within 28 days or 5 terminal half-livesbefore
screening, or participation in an interventional study of a non-investigational treatment from screening through end of study participation.
9. Use of restricted medication within specified duration before the first dose of study drug.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified