An extension study to assess the long-term safety and efficacy of a triple combination therapy of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in children with cystic fibrosis who are at least 1 year old.
Summary description of the study
The aim of this extension study (VX22-445-123) is to assess the long-term safety and efficacy of a triple combination therapy of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in children with cystic fibrosis who are at least 1 year old. Participation in this study lasts just under two years. All participants receive the investigational drug, and both the parents/legal guardians of the participating children and the investigator know the administered dose. The insights gained from this study may be helpful in the future for the treatment of cystic fibrosis or other diseases and deepen scientific understanding.
(BASEC)
Intervention under investigation
In this study, the term "investigational drug" refers to the triple combination of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA). ELX/TEZ/IVA is approved in some countries for use in certain patients with cystic fibrosis, and the researchers believe it may also be beneficial for younger age groups.
Even though the investigational drug was used in the main study (VX22-445-122), it is considered an investigational drug for this age group, meaning it has not yet been approved by some health authorities and is still being studied regarding long-term safety and efficacy for patients in this age group.
Participants in this study will receive one of four possible doses of the investigational drug based on their body weight. The dose may be adjusted if body weight changes during the treatment period.
Participation in the study lasts approximately 100 weeks (up to 96 weeks for the study treatment period and up to 4 weeks for the safety follow-up period).
Overall, participants, accompanied by parents, will come to the clinic at least 11 times over a period of 23 months. A study visit may last from 30 minutes to 4 hours. After 25 months, participation in the study is completed.
(BASEC)
Disease under investigation
This study (extension study, VX22-445-123) targets children who are at least 1 year old, suffer from cystic fibrosis, and have participated in the main study (VX22-445-122).
(BASEC)
1. The parents or legal guardian of the participating child have signed an informed consent form to participate. 2. At the discretion of the investigator, the parents or guardian must be able to understand the study requirements, restrictions, and instructions, and ensure that the child adheres to the study requirements and that the study is likely to be completed as planned. 3. The informed consent form was not revoked for the main study (VX22-445-122). (BASEC)
Exclusion criteria
1. A disease or clinical condition has occurred in the participating child that could distort the study results or pose an additional risk during the administration of the investigational drug(s), such as liver cirrhosis, solid organ or hematological transplantation, cancer. 2. A drug intolerance occurred in the participating child in the main study VX22-445-122 that would expose the participating child to an additional risk. 3. Current participation in another clinical study other than the main study VX22-445-122. 4. Poor adherence to the investigational drug and/or does not follow the procedures in the main study VX22-445-122 according to the investigator's opinion. (BASEC)
Trial sites
Bern, Zurich
(BASEC)
Sponsor
Vertex Pharmaceuticals (CH) GmbH
(BASEC)
Contact
Contact Person Switzerland
Prof. Dr. Alexander Möller
+41442667079
alexander.moeller@clutterkispi.uzh.chUniversitäts-Kinderspital Zürich, Pädiatrie, pädiatrische Pneumologie, Schlafmedizin (SSSSC)
(BASEC)
Scientific Information
not available
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Zurich
(BASEC)
Date of authorisation
29.10.2024
(BASEC)
ICTRP Trial ID
not available
Official title (approved by ethics committee)
Eine offene Phase-III-Studie zur Beurteilung der Langzeitsicherheit und -wirksamkeit von Elexacaftor/Tezacaftor/Ivacaftor bei Mukoviszidose-Patienten ab 12 Monaten (BASEC)
Academic title
not available
Public title
not available
Disease under investigation
not available
Intervention under investigation
not available
Type of trial
not available
Trial design
not available
Inclusion/Exclusion criteria
not available
not available
Primary and secondary end points
not available
not available
Registration date
not available
Incorporation of the first participant
not available
Secondary sponsors
not available
Additional contacts
not available
Secondary trial IDs
not available
Results-Individual Participant Data (IPD)
not available
Further information on the trial
not available
Results of the trial
Results summary
not available
Link to the results in the primary register
not available