Phase 2a Study of VX-147 in Adults With APOL1-mediated FSGS

• Research type

  Research Study

• Full title

  A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis.

• IRAS ID

  281304

• Contact name

  Kate Bramham

• Contact email

  kate.bramham@kcl.ac.uk

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2020-000185-42

• Duration of Study in the UK

  0 years, 12 months, 0 days

• Research summary

  This is a Phase 2a a single-arm, open-label, 2-part study of VX 147 in Adults With APOL1 mediated Focal Segmental Glomerulosclerosis( FSGS), in male and female subject aged 18 to 60 years old.
  In Part A, subjects will receive VX 147 at a dosage of 15 mg once daily for 2 weeks and VX 147 at a dosage of 45 mg once daily for 11 weeks. Part A will be enrolled in 2 cohorts: Cohort 1 and an optional Cohort 2. Cohort 1 includes at least 10 subjects with UPCR ≥3 g/g and <10 g/g who are not taking systemic corticosteroid or other immunosuppressants. Cohort 2 includes approximately 10 subjects with UPCR ≥2 g/g and <10 g/g, and these subjects are permitted to take a stable low dose of systemic corticosteroid.

  After completing Part A, subjects will be followed for up to 12 weeks in Part B (optional) for evaluation of proteinuria off treatment.
  All subjects will complete a Safety Follow-up Visit (SFUV) at 28 (± 7) days after the last dose of study drug.
  There are about 15 study visits; some of these visits may be conducted at the participant’s home by a home health instructor.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  20/LO/0639

• Date of REC Opinion

  1 Jun 2020

• REC opinion

  Further Information Favourable Opinion