Clinical trial • Phase III • Respiratory

TEZACAFTOR, ELEXACAFTOR, IVACAFTOR for Cystic fibrosis

Phase III trial of TEZACAFTOR, ELEXACAFTOR, IVACAFTOR for Cystic fibrosis. open-label. 35 participants.

Overview

Trial Therapeutic Area: Respiratory
Trial Disease: Cystic fibrosis
Trial Stage: Phase III
Drug Modality: Small molecule
Paediatric Trial: Yes
Orphan Drug: Yes

Key dates

Initial CTIS Submission Date: 19-07-2024
First CTIS Authorization Date: 21-10-2024

Trial design

open-label Phase III trial in Denmark, Netherlands, Germany.

Open Label: Yes
Target Sample Size: 35

Eligibility

Recruits 35 paediatric patients.

Vulnerable Population: The study includes paediatric subjects (from 12 months of age and older). Consent must be provided by the subject’s legally appointed and authorized representative who will sign and date the informed consent form (ICF). The legal representative/guardian must be able to understand protocol requirements, restrictions, and instructions and be able to ensure that the subject will comply with and is likely to complete the study as planned.

Inclusion criteria

{"criterion_text":"- Subject’s legally appointed and authorized representative will sign and date the informed consent form (ICF)."}
{"criterion_text":"- As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions, and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned."}
{"criterion_text":"- Did not withdraw consent from the parent study."}
{"criterion_text":"- Meets at least 1 of the following criteria: • Completed study drug treatment in the parent study, or • Had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of the parent study."}
{"criterion_text":"- Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation."}

Exclusion criteria

{"criterion_text":"- 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. This includes, but is not limited to, the following: • Clinically significant liver cirrhosis with or without portal hypertension • Solid organ or hematological transplantation • Cancer"}
{"criterion_text":"- History of drug intolerance in the 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)."}
{"criterion_text":"- Current participation in an investigational drug trial other than the 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."}
{"criterion_text":"- History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study as deemed by the investigator."}
{"criterion_text":"- Use of restricted medication, as defined in Table 9-2, unless subject is on a study drug interruption at the time of rollover."}
{"criterion_text":"- The subject or 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 the site."}

Endpoints

Primary endpoints

{"endpoint_text":"- Safety and tolerability assessments as determined by adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry","definition_or_measurement_approach":"Assessment determined by adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry"}

Secondary endpoints

{"endpoint_text":"- Absolute change in sweat chloride (SwCl)","definition_or_measurement_approach":"Absolute change in sweat chloride (SwCl) (measurement method not detailed in provided record)"}

Recruitment

Planned Sample Size: 35
Recruitment Window Months: 33
Consent Approach: Informed consent is obtained from the subject’s legally appointed and authorized representative (ICF must be signed and dated by the representative). The protocol requires the legal representative/guardian to understand protocol requirements and to ensure the subject's compliance. Country-specific subject information and informed consent forms are provided (document titles indicate available languages/local versions: Denmark - Danish; Netherlands - Dutch; Germany - German). Separate parent/guardian ICF documents and ICF tools are provided per country.

Geography

Total Number Of Sites: 5
Total Number Of Participants: 15

Denmark

Earliest CTIS Part Ii Submission Date: 09-10-2024
Latest Decision Or Authorization Date: 15-05-2025
Processing Time Days: 218
Number Of Sites: 1
Number Of Participants: 3

Sites

Site Name: Rigshospitalet
Department Name: CF Center, Copenhagen and Pediatric Pulmonary Unit Pediatric Department 5003
Contact Person Name: Marianne Skov
Contact Person Email: Marianne.Skov@regionh.dk
Number Of Participants: 3

Netherlands

Earliest CTIS Part Ii Submission Date: 20-09-2024
Latest Decision Or Authorization Date: 15-05-2025
Processing Time Days: 237
Number Of Sites: 1
Number Of Participants: 3

Sites

Site Name: Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name: Department of Pediatrics, division of Respiratory Medicine and Allergology
Contact Person Name: Hettie Janssens
Contact Person Email: h.janssens@erasmusmc.nl
Number Of Participants: 3

Germany

Earliest CTIS Part Ii Submission Date: 29-08-2024
Latest Decision Or Authorization Date: 15-05-2025
Processing Time Days: 259
Number Of Sites: 3
Number Of Participants: 9

Sites

Site Name: Charite Universitaetsmedizin Berlin KöR
Department Name: Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine
Contact Person Name: Marcus Mall
Contact Person Email: marcus.mall@charite.de

Site Name: Universitaetsklinikum Essen AöR
Department Name: Pediatric Pulmonology
Contact Person Name: Florian Stehling
Contact Person Email: Florian.Stehling@uk-essen.de

Site Name: Medizinische Hochschule Hannover
Department Name: Pediatric Pneumology, Allergology and Neonatology
Contact Person Name: Anna-Maria Dittrich
Contact Person Email: Dittrich.Anna-Maria@mh-hannover.de

Sponsor

Primary sponsor

Full Name: Vertex Pharmaceuticals Inc.
Organisation Type: Pharmaceutical company
Country Of Registered Address: United States

Investigational products

Investigational Product Name: VX-445/VX-661/VX-770 fixed-dose combination granules
Active Substance: TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 100 mg

Investigational Product Name: VX-770 75mg
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 75 mg

Investigational Product Name: VX-770 granules
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 59.5 mg

Investigational Product Name: Kaftrio 75 mg/50 mg/100 mg granules in sachet
Active Substance: TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=2
Orphan Designation: Yes
Maximum Dose: 100 mg

Investigational Product Name: Kalydeco 50 mg granules in sachet
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=2
Orphan Designation: Yes
Maximum Dose: 50 mg

Investigational Product Name: VX-445/VX-661/VX-770 fixed-dose combination granules
Active Substance: TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 80 mg

Investigational Product Name: Kaftrio 60 mg/40 mg/80 mg granules in sachet
Active Substance: TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=2
Orphan Designation: Yes
Maximum Dose: 80 mg

Investigational Product Name: VX-770 50mg
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 50 mg

Investigational Product Name: Kalydeco 59.5 mg granules in sachet
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=2
Orphan Designation: Yes
Maximum Dose: 59.5 mg

Investigational Product Name: Kalydeco 25 mg granules in sachet
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=2
Orphan Designation: Yes
Maximum Dose: 25 mg

Investigational Product Name: Ivacaftor 25 mg granules
Active Substance: IVACAFTOR
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 25 mg

Combination Treatment: Yes

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