R3R01 in Alport Syndrome Patients and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

• Research type

  Research Study

• Full title

  A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

• IRAS ID

  1005197

• Contact name

  Guido Magni, MD, PhD

• Sponsor organisation

  River 3 Renal, Corporation

• Eudract number

  2021-004192-13

• Clinicaltrials.gov Identifier

  NCT05267262

• Research summary

  R3R01 is being developed for the treatment of Alport syndrome (AS) and Focal Segmental Glomerulosclerosis (FSGS). AS and FSGS are both a type of kidney disease. AS results from a defect in the genes and can result in kidney failure, hearing loss, and eye abnormalities. FSGS results in scarring or hardening (sclerosis) in the kidney. In both AS and FSGS, animal studies show that the accumulation of fat in the kidney cells can cause injury to the kidney cells.
  R3R01 works by increasing the activity of a protein in the body, which helps in regulating fat levels in the kidney. There is evidence from testing in animals that R3R01 may decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney.
  R3R01 is an experimental drug. Experimental means that the study drug is currently being tested and is not approved for use by any health authorities around the world.
  The main purpose of this study is to check how safe the study drug is and how well participants handles taking it.
  The study will also check:
  • if the study drug works to improve participants kidney function
  • whether the study drug has an impact on participants daily life
  • the amount of the study drug in participants blood over a period of time (called pharmacokinetics)
  Participants are being invited to join this study if:
  • Participants are 12 years of age or older (up to 75 years of age if they have FSGS)
  • Participants have AS or FSGS

  Up to 20 participants with AS and up to 30 participants with FSGS will take part in the study. This study is being conducted at about 30 sites in the United States (USA) and Europe. The study is sponsored by River 3 Renal Corp.

• REC name

  North West - Greater Manchester South Research Ethics Committee

• REC reference

  22/NW/0173

• Date of REC Opinion

  17 Aug 2022

• REC opinion

  Further Information Favourable Opinion