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)
Zusammenfassung der Studie
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)
Untersuchte Intervention
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)
Untersuchte Krankheit(en)
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)
Ausschlusskriterien
Patients who have discontinued the parent study or who have not participated in any parent studies are excluded from participation. (BASEC)
Studienstandort
Zürich
(BASEC)
Sponsor
Vertex Pharmaceuticals Incorporated, Boston, USA Vertex Pharmaceuticals GmbH, Zug, Switzerland
(BASEC)
Kontakt für weitere Auskünfte zur Studie
Kontaktperson Schweiz
Dr. Sabine Albrecht, Associate Medical Director
+41 (0)41 560 05 00
sabine_albrecht@cluttervrtx.comVertex Pharmaceutical GmbH
(BASEC)
Wissenschaftliche Auskünfte
nicht verfügbar
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Zürich
(BASEC)
Datum der Bewilligung durch die Ethikkommission
10.01.2023
(BASEC)
ICTRP Studien-ID
NCT05444257 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
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)
Wissenschaftlicher Titel
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)
Öffentlicher Titel
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy (ICTRP)
Untersuchte Krankheit(en)
Cystic Fibrosis (ICTRP)
Untersuchte Intervention
Drug: VX-121/TEZ/D-IVA (ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Ein-/Ausschlusskriterien
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)
nicht verfügbar
Primäre und sekundäre Endpunkte
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)
Registrierungsdatum
nicht verfügbar
Einschluss des ersten Teilnehmers
nicht verfügbar
Sekundäre Sponsoren
nicht verfügbar
Weitere Kontakte
nicht verfügbar
Sekundäre IDs
2021-000713-17, 2024-514173-22-00, VX20-121-104 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://clinicaltrials.gov/ct2/show/NCT05444257 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar