Table of Contents

• Clinical trial overview
• Who the trial is for
• Study design and phase
• Main endpoint
• What the results are meant to show

Clinical trial overview

One authorised study is investigating whether RNA, (AM-SP-UM-CM-AM-AM-CM-UM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)A-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)C-UM-GM-UM-AM-AM-UM-AM-AM-AM-GM-CM-AM-GM-CM-CM-GM-[2′-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-[2′-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-[2′-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-GM-GM-CM-UM-GM-CM), COMPLEX WITH RNA ([4′-DE(HYDROXYMETHYL)-4′-[(HYDROXYMETHOXYPHOSPHINYL)METHOXY]]UM-SP-(2′-DEOXY-2′-FLUORO)U-SP-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-(2′-DEOXY-2′-FLUORO)U-UM-(2′-DEOXY-2′-FLUORO)A-CM-AM-(2′-DEOXY-2′-FLUORO)G-GM-UM-GM-(2′-DEOXY-2′-FLUORO)A-GM-UM-UM-GM-AM-UM-SP-GM-SP-GM) is effective and safe in adults with antibody-mediated rejection after kidney transplantation.

Who the trial is for

The study is for adults with antibody-mediated rejection after kidney transplantation. The summary also says the participants may have active or chronic active antibody-mediated rejection, which means the rejection can be happening now or continuing over time.

Study design and phase

This is a Phase 2 interventional study. In a Phase 2 trial, researchers look at early signs of benefit and safety in a smaller group of people.

The trial compares the study treatment with vialed saline placebo, which is an inactive treatment used for comparison. The listed enrollment is 50 participants.

Main endpoint

The main endpoint is biopsy-proven histologic resolution at Week 52. A biopsy is a small sample of tissue, and histologic resolution means the tissue looks healed or improved under the microscope.

This endpoint matters because it shows whether the kidney transplant rejection has improved in a way that can be seen directly in the kidney tissue.

What the results are meant to show

The brief summary says the study is designed to compare ALXN2030 with placebo for biopsy-proven histologic resolution in participants with active or chronic active antibody-mediated rejection at Week 52.

For patients, this means the trial is not only checking whether the treatment is tolerated, but also whether it may help the transplanted kidney recover better on biopsy after about one year.