Informazioni generali
  • Categoria della malattia Malattie genetiche (BASEC)
  • Fase dello studio Phase 3 (ICTRP)
  • Stato di reclutamento reclutamento completato (BASEC/ICTRP)
  • Luogo dello studio
    Zurigo
    (BASEC)
  • Responsabile dello studio Dr. Sabine Albrecht, Associate Medical Director sabine_albrecht@vrtx.com (BASEC)
  • Fonte dati BASEC: Importato da 15.04.2025 ICTRP: Importato da 23.08.2024
  • Ultimo aggiornamento 15.04.2025 14:36
HumRes62494 | SNCTP000005296 | BASEC2022-01779 | NCT05444257

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)

  • Categoria della malattia Malattie genetiche (BASEC)
  • Fase dello studio Phase 3 (ICTRP)
  • Stato di reclutamento reclutamento completato (BASEC/ICTRP)
  • Luogo dello studio
    Zurigo
    (BASEC)
  • Responsabile dello studio Dr. Sabine Albrecht, Associate Medical Director sabine_albrecht@vrtx.com (BASEC)
  • Fonte dati BASEC: Importato da 15.04.2025 ICTRP: Importato da 23.08.2024
  • Ultimo aggiornamento 15.04.2025 14:36

Descrizione riassuntiva dello studio

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)

Intervento studiato

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)

Malattie studiate

cystic fibrosis

(BASEC)

Criteri di partecipazione
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)

Criteri di esclusione
Patients who have discontinued the parent study or who have not participated in any parent studies are excluded from participation. (BASEC)

Luogo dello studio

Zurigo

(BASEC)

Australia, Austria, Belgium, Canada, Czechia, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Netherlands, New Zealand, Norway, Poland, Portugal, Spain, Sweden, Switzerland, United Kingdom, United States (ICTRP)

Sponsor

Vertex Pharmaceuticals Incorporated, Boston, USA Vertex Pharmaceuticals GmbH, Zug, Switzerland

(BASEC)

Contatto per ulteriori informazioni sullo studio

Persona di contatto in Svizzera

Dr. Sabine Albrecht, Associate Medical Director

+41 (0)41 560 05 00

sabine_albrecht@vrtx.com

Vertex Pharmaceutical GmbH

(BASEC)

Informazioni scientifiche

non disponibile

Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)

Commissione etica Zurigo

(BASEC)

Data di approvazione del comitato etico

10.01.2023

(BASEC)


ID di studio ICTRP
NCT05444257 (ICTRP)

Titolo ufficiale (approvato dal comitato etico)
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)

Titolo accademico
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)

Titolo pubblico
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy (ICTRP)

Malattie studiate
Cystic Fibrosis (ICTRP)

Intervento studiato
Drug: VX-121/TEZ/D-IVA (ICTRP)

Tipo di studio
Interventional (ICTRP)

Disegno dello studio
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)

Criteri di inclusione/esclusione
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 disponibile

Endpoint primari e secondari
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)

Data di registrazione
non disponibile

Inclusione del primo partecipante
non disponibile

Sponsor secondari
non disponibile

Contatti aggiuntivi
non disponibile

ID secondari
2021-000713-17, 2024-514173-22-00, VX20-121-104 (ICTRP)

Risultati-Dati individuali dei partecipanti
non disponibile

Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT05444257 (ICTRP)

Risultati dello studio

Riepilogo dei risultati

non disponibile

Link ai risultati nel registro primario

non disponibile