Study to assess the long-term safety and long-term efficacy of Elexacaftor/Tezacaftor/Ivacaftor in participants without F508del mutation

Identifiant de l'essai ICTRP
EUCTR2021-005914-33 (ICTRP)

Titre officiel (approuvé par le comité d'éthique)
non disponible

Titre académique
A Phase 3 Open-label Study Evaluating the Longterm Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes - Study to Evaluate Elx/Tez/Iva Long-term Safety and Efficacy in Subjects Without F508del (ICTRP)

Titre public
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor Long-term Safety and Efficacy in Subjects Without F508del (ICTRP)

Maladie en cours d'investigation
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] (ICTRP)

Intervention étudiée

Trade Name: Kaftrio
Product Name: 100mg ELX/50mg TEZ /75mg IVA
Product Code: VX-445/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Elexacaftor
CAS Number: 2216712-66-0
Current Sponsor code: VX-445
Other descriptive name: ELX
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
INN or Proposed INN: Tezacaftor
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Other descriptive name: TEZ
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
INN or Proposed INN: Ivacaftor
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Other descriptive name: IVA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Trade Name: Kalydeco
Product Name: 150mg Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Ivacaftor
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Other descriptive name: IVA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Trade Name: Kalydeco
Product Name: 75-mg Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Ivacaftor
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Other descriptive name: IVA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Trade Name: Kaftrio
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA
Product Code: VX-445/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Elexacaftor
CAS Number: 2216712-66-0
Current Sponsor code: VX-445
Other descriptive name: ELX
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
INN or Proposed INN: Tezacaftor
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Other d (ICTRP)

Type d'essai
Interventional clinical trial of medicinal product (ICTRP)

Plan de l'étude
Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1 (ICTRP)

Critères d'inclusion/exclusion
Gender:
Female: yes
Male: yes

Inclusion criteria:
1. Subject (or the subject?s 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 (as applicable), and other study procedures.
- For subjects <18 years of age: as judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
3. Did not withdraw consent from a parent study.
4. Part A: Meets at least 1 of the following criteria:
- Completed study drug treatment in a parent study.
- Had study drug interruption(s) in a parent study, but completed study visits up to the last scheduled visit of the Treatment Period of a parent study.
Part B: Meets at least 1 of the following criteria:
- Completed study drug treatment in Part A
- Had study drug interruption(s) in Part A, but completed study visits up to the last scheduled visit of the Treatment Period of Part A
5. Willing to remain on a stable CF treatment regimen (other than CFTR modulators, as defined in Section 9.5) through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 120
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
(ICTRP)

Exclusion criteria:
1. History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
2. History of drug intolerance in a parent study that would pose an additional risk to the subject. (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
3. Pregnant and nursing females. Females of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Day 1 Visit (in Part A and Part B) before receiving the first dose of study drug.
4. Current participation in an investigational drug trial (other than a parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.


Critères d'évaluation principaux et secondaires
Main Objective: Part A and B: To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
;Secondary Objective: Part A Only: To evaluate the long-term efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
;Primary end point(s): Part A and B:
Safety and tolerability based on AEs, clinical laboratory values, ECGs, vital signs, and pulse oximetry;Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit.
(ICTRP)

Secondary end point(s): Part A ONLY:
- Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
- Absolute change from baseline in SwCl
- Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score.
- Absolute change from baseline in body mass index (BMI)
- Absolute change from baseline in weight
- Number of pulmonary exacerbations (PEx)
Other Endpoints
- Absolute change from baseline in BMI z-score (subjects =20 years of age)
- Absolute change from baseline in weight z-score (subjects =20 years of age);Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit. (ICTRP)

Date d'enregistrement
16.05.2022 (ICTRP)

Inclusion du premier participant
non disponible

Sponsors secondaires
non disponible

Contacts supplémentaires
Clinical Trials and Medical Info, medicalinfo@vrtx.com, +18776348789, Vertex Pharmaceuticals Incorporated (ICTRP)

ID secondaires
VX21-445-125, 2021-005914-33-DE (ICTRP)

Résultats-Données individuelles des participants
non disponible

Informations complémentaires sur l'essai
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2021-005914-33 (ICTRP)