Full name

A Randomised, Double Blind, Placebo-controlled, Single Ascending Dose, Phase 1a/1b Multi-centre Study in Healthy Participants and Participants With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of GSK4771261

NCT Number
NCT06734234
Primary Endpoints

Primary:

  • Part A: Number of Participants with Adverse events (AEs). From Baseline (Day 1) up to Day 182 (End of study [EoS])
  • Part B: Number of Participants with AEs. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Number of Participants with Serious Adverse Events (SAEs). From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with SAEs. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Number of Participants with Clinically Significant Changes in Blood Laboratory Values. From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with Clinically Significant Changes in Blood Laboratory Values. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Number of Participants with Clinically Significant Changes in Urine Laboratory Values. From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with Clinically Significant Changes in Urine Laboratory Values. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Number of Participants with Clinically Significant Changes in Vital Signs. From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with Clinically Significant Changes in Vital Signs. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Number of Participants with Clinically Significant Changes in 12-lead Electrocardiogram (ECG). From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with Clinically Significant Changes in 12-lead ECG. From Baseline (Day 1) up to Day 182 (EoS)

Secondary:

  • Part A: Number of Participants with Anti-drug Antibodies for GSK4771261. From Baseline (Day 1) up to Day 182 (EoS)
  • Part B: Number of Participants with Anti-drug Antibodies for GSK4771261. From Baseline (Day 1) up to Day 182 (EoS)
  • Part A: Area under the concentration-time curve to the end of the dosing period AUC (0-tau) of GSK4771261. Up to Day 182
  • Part A: Area under the concentration-time curve to infinity AUC (0-inf) of GSK4771261. Up to Day 182
  • Part B: AUC (0-tau) of GSK4771261. Up to Day 182
  • Part B: AUC (0-inf) of GSK4771261. Up to Day 182
  • Part A: Maximum Plasma Concentration (Cmax) of GSK4771261. Up to Day 182
  • Part B: Cmax of GSK4771261. Up to Day 182
Order
0
Disease
ADPKD
Menu title
A Study to Evaluate the Safety, Tolerability, and Effects on Blood and Urine Markers of Single Ascending Dose of GSK4771261 in Healthy Participants and Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Version
ASN 2025
Phase
1
Status
Recruiting
Compound name
GSK4771261
MoA
mAb against novel kidney target
Unmet Burden Fact
  • ADPKD is the most common genetic kidney disease worldwide. In the US, the estimated number of persons with clinically recognized ADPKD is 140,000-240,000¹ 
  • ADPKD is the 4th most-common global cause for kidney replacement therapy² with approximately 50% of individuals with ADPKD developing ESKD by age 62¹
  • Randomized controlled trials tailored to the ADPKD population remain a critical need³
Unmet Burden Fact Reference

1. Chebib FT, et al. JAMA 2025; 333(19): 1708-1719; 2. KDIGO 2025 Clinical Practice Guideline for the Evaluation, Management, and Treatment of ADPKD. Kidney Int. 2025;107(2S): S1-S239; 3. Fang, C et al. Curr Opin Nephrol Hyperten 2025, 34:368-374

Study Description

This study will assess safety, tolerability, pharmacokinetics and pharmacodynamics of GSK4771261 in Healthy Participants (Part A)  and Participants with Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Part B)