An open phase III study to assess the long-term safety and efficacy of the VX-121 combination therapy in participants with cystic fibrosis (CF)
Résumé de l'étude
This study is being conducted to learn more about the safety and efficacy of the combination of VX-121/TEZ/D-IVA in participants with CF. Male and female patients with cystic fibrosis who are at least 12 years old, have successfully completed one of the previous Vertex studies (including 121-102 or 121-103), and meet the inclusion and exclusion criteria can participate. Patients will be treated in this study with the same dosing of the morning medication VX-121/TEZ/D-IVA as in the previous study. The study lasts a total of up to 100 weeks, of which up to 96 weeks are treatment phase.
(BASEC)
Intervention étudiée
The investigational drug (20 mg VX-121/100 mg TEZ/250 mg D-IVA) is taken once daily at approximately the same time: two tablets.
(BASEC)
Maladie en cours d'investigation
cystic fibrosis
(BASEC)
For Part A: Adults, adolescents, and children who have successfully completed one of the parent studies VX20-121-102 or VX20-121-103. For Part B: Adults, adolescents, and children who have successfully completed treatment with VX-121/TEZ/D-IVA in Part A. (BASEC)
Critères d'exclusion
Patients who have discontinued the parent study or who have not participated in any parent studies are excluded from participation. (BASEC)
Lieu de l’étude
Zurich
(BASEC)
Sponsor
Vertex Pharmaceuticals Incorporated, Boston, USA Vertex Pharmaceuticals GmbH, Zug, Switzerland
(BASEC)
Contact pour plus d'informations sur l'étude
Personne de contact en Suisse
Dr. Sabine Albrecht, Associate Medical Director
+41 (0)41 560 05 00
sabine_albrecht@cluttervrtx.comVertex Pharmaceutical GmbH
(BASEC)
Informations scientifiques
non disponible
Nom du comité d'éthique approbateur (pour les études multicentriques, uniquement le comité principal)
Commission cantonale de Zurich
(BASEC)
Date d'approbation du comité d'éthique
10.01.2023
(BASEC)
Identifiant de l'essai ICTRP
NCT05444257 (ICTRP)
Titre officiel (approuvé par le comité d'éthique)
A Phase 3, Open-label Study Evaluating the Longterm Safety and Efficacy of VX-121/TEZ/D-IVA Combination Therapy in Subjects With Cystic Fibrosis (BASEC)
Titre académique
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121/TEZ/D-IVA Combination Therapy in Subjects With Cystic Fibrosis (ICTRP)
Titre public
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy (ICTRP)
Maladie en cours d'investigation
Cystic Fibrosis (ICTRP)
Intervention étudiée
Drug: VX-121/TEZ/D-IVA (ICTRP)
Type d'essai
Interventional (ICTRP)
Plan de l'étude
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Critères d'inclusion/exclusion
Gender: All
Maximum age: N/A
Minimum age: 12 Years
Key Inclusion Criteria:
- Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080)
and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study
but did not permanently discontinue study drug, and completed study visits up to the
last scheduled visit of the Treatment Period in the parent study
- Part B: Completed study drug treatment in Part A; or had study drug interruption(s)
in Part A, but did not permanently discontinue study drug, and completed study
visits up to the last scheduled visit of the Treatment Period of Part A
Key Exclusion Criteria:
- History of drug intolerance in a parent study
- Pregnant or breast-feeding females at the time of enrollment in Part A
Other protocol defined Inclusion/Exclusion criteria may apply. (ICTRP)
non disponible
Critères d'évaluation principaux et secondaires
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (ICTRP)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1);Absolute Change From Baseline in Sweat Chloride (SwCl);Number of Pulmonary Exacerbations (PEx) (ICTRP)
Date d'enregistrement
non disponible
Inclusion du premier participant
non disponible
Sponsors secondaires
non disponible
Contacts supplémentaires
non disponible
ID secondaires
2021-000713-17, 2024-514173-22-00, VX20-121-104 (ICTRP)
Résultats-Données individuelles des participants
non disponible
Informations complémentaires sur l'essai
https://clinicaltrials.gov/ct2/show/NCT05444257 (ICTRP)
Résultats de l'essai
Résumé des résultats
non disponible
Lien vers les résultats dans le registre primaire
non disponible