Phase 1/2 study of PS-002, a Gene Therapy for the Treatment of IgA Nephropathy

• Research type

  Research Study

• Full title

  A Phase 1/2 Multicenter, Open Label, Two-part Study (Single Ascending Dose [Part 1], and Dose Expansion [Part 2]) to Evaluate Safety, Tolerability and Efficacy of PS-002, a Gene Therapy for the Treatment of Adult Participants with Primary IgA Nephropathy

• IRAS ID

  1011740

• Contact name

  Robert Morgan

• Contact email

  r.morgan@purespringtx.com

• Sponsor organisation

  Purespring Therapeutics Ltd.

• Research summary

  This study is being completed to find out if a new investigational product called PS-002, which is considered a type of gene therapy is safe and an effective treatment for people with Immunoglobulin A (IgA) nephropathy.
  IgA nephropathy is a kidney condition that occurs when the body makes abnormal IgA (an antibody) which builds up in the kidneys due to their abnormal structure and function, causing inflammation and damage to kidneys over time. Gene therapy can be used to treat a disease or condition at the cell level by changing or fixing how the cell functions.

  In this study, researchers are delivering instructions that produces the Complement Factor I (CFI) protein in specific kidney cells called “podocytes”, enhancing CFIs impact. As a result, the podocytes will have the instructions to build more CFI protein, helping to reduce inflammation and damage.

  All participants will receive a one-time infusion of PS-002, administered directly to each kidney through the renal arteries called a renal infusion.

  This is a first in human study and has two parts:
  - Part 1: up to 12 participants will be included to find the best dose of PS-002 that works without causing harmful side effects.
  - Part 2: up to 20 participants will be included to collect more information once the most effective and safe dose is found in Part 1.

  Participants will only participate in either Part 1 or Part 2 and will be in the study for up to 56 weeks (with up to 16 study visits). This includes up to 8 weeks screening, an in-patient stay of 4 days (one day pre-infusion, the day of infusion, plus 2 full days following the PS-002 administration), and 48 weeks of follow-up.
  Assessments will include kidney biopsy, kidney imaging studies, physical examinations, ECGs, health questionnaires, blood and urine tests.

  At the end of the study, participants will be invited to join a separate optional long-term follow-up study for an additional 4 years.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  25/LO/0452

• Date of REC Opinion

  22 Jul 2025

• REC opinion

  Further Information Favourable Opinion