VX25-828-101

• Research type

  Research Study

• Full title

  A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-828/Deutivacaftor With and Without Tezacaftor in Subjects Aged 18 Years and Older With Cystic Fibrosis

• IRAS ID

  1013749

• Contact name

  Ricky Radia

• Contact email

  Ricky_Radia@vrtx.com

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Research summary

  There will be 4 parts to this study. This study is being done to learn more about the safety and effectiveness of different doses of VX-828 in the combination of VX-828/D-IVA with (Parts 1 and 3) or without (Parts 2 and 4) TEZ in people with Cystic Fibrosis (CF) caused by specific change in their genes that causes CF: F/MF genotype (Parts 1 and 2) or F/F genotype (Parts 3 and 4).
  
  VX-828/TEZ/D-IVA is an investigational drug; “investigational” means the drug is not approved by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA) in the European Union, the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK, or health authorities in other countries and is still being tested for safety and efficacy. The triple combination of VNZ/TEZ/D-IVA, which is used in the study, is approved for use in some countries for certain patients with CF.

  Male and female participants with CF that are 18 years of age and older will be invited to participate in the study. Approximately 152 participants: 56 in Part 1, up to 64 in Part 2, 16 in Part 3, and 16 in Part 4. The number of participants has been carefully determined to be sure that the data collected on safety and effectiveness of VX-828/D-IVA with or without TEZ is reliable.

  Participation will last for approximately 108 days. Approximately 167 mL (9 tablespoons) of blood will be taken for Parts 1 and 2 and approximately 142 mL (8 tablespoons) of blood will be taken for Parts 3 and 4. The participants will attend site around 12 times for Parts 1 and 2 and around 8 times for Parts 3 and 4 and will undergo the following assessments:
  • Medical history
  • Physical examination
  • Vital signs
  • Pulse oximetry
  • Electrocardiogram (ECG or heart tracing)
  • Questionnaires
  • Spirometry
  • Sweat Chloride
  • Blood samples
  • Urine samples
  • Pregnancy/hormone test
  • Genotype sample
  • Blood samples to measure CF drug concentrations

• REC name

  East of England - Cambridge East Research Ethics Committee

• REC reference

  26/EE/0074

• Date of REC Opinion

  5 Jun 2026

• REC opinion

  Further Information Favourable Opinion