A Study to Evaluate the Safety, Tolerability, and Effects on Blood and Urine Markers of Single As… (NCT06734234) | Clinical Trial Compass
Active — Not RecruitingPhase 1
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
United States, Belgium, Canada86 participantsStarted 2024-12-11
Plain-language summary
This is a study where a new drug, called GSK4771261 is being tested. Neither the study doctors, study staff or participants will be aware of what treatment is being given. Part A is testing the new study treatment on healthy people. This is to see if it's safe, what it does to the body, and how the body's defense system responds to it. Part B is similar, but the study treatment will be given to people who have a kidney disease called autosomal dominant polycystic kidney disease (ADPKD).
Who can participate
Age range
25 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
For Part A
* Body weight greater than or equal to (\>=) 45 kilograms (kg) and basal metabolic index (BMI) within the range 19.5 to 32 kilograms per square meters (kg/m\^2), inclusive
* Capable of giving signed informed consent,
* Participants who are overtly healthy as determined by medical evaluation by the investigator or a medically qualified designee based on medical history, physical examination, laboratory tests, and cardiac monitoring
* Women must be of non-childbearing potential
For Part B (planned Cohorts 1-3 and optional cohorts 4-5):
* Body weight \>=45 kg and BMI within the range 19.5 to 32 kg/m\^2, inclusive.
* Capable of giving signed informed consent, Confirmed diagnosis of Autosomal dominant polycystic kidney disease (ADPKD) by either applicable guidelines and/or genetic and imaging screening assessments
* Participants diagnosed with ADPKD may have complications or comorbidities directly related to ADPKD but should be otherwise healthy as determined by the investigator or medically qualified designee based on a medical evaluation including medical history, physical examination, laboratory tests clinical testing
* Confirmation of known ADPKD causal genetic mutation(s) at the Polycystic kidney disease (PKD)1 and/or PKD2 loci based on genetic testing at screening and/or existing genetic information collected during the participant's routine clinical care for ADPKD from a genetic testing provider that, in the judgement of the investigator, …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Part A: Number of Participants with Adverse events (AEs)
Timeframe: From Baseline (Day 1) up to Day 182 (End of study [EoS])
2
Part B: Number of Participants with AEs
Timeframe: From Baseline (Day 1) up to Day 182 (EoS)
3
Part A: Number of Participants with Serious Adverse Events (SAEs)
Timeframe: From Baseline (Day 1) up to Day 182 (EoS)
4
Part B: Number of Participants with SAEs
Timeframe: From Baseline (Day 1) up to Day 182 (EoS)
5
Part A: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Timeframe: From Baseline (Day 1) up to Day 182 (EoS)
6
Part B: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Timeframe: From Baseline (Day 1) up to Day 182 (EoS)
7
Part A: Number of Participants with Clinically Significant Changes in Urine Laboratory Values