Kidney DiseasePhase 1NCT06734234
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
A Phase 1 study testing GSK4771261 in people with Kidney Disease, run by GlaxoSmithKline at 23 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 25 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Kidney Disease
- What is being tested?
- GSK4771261
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 86 participants
- Where
- 23 sitesin 8 countries
- Study dates
- 11 Dec 2024to 23 Jul 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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):
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- 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, is clinically valid.
- Mayo imaging classification groups 1C, 1D or 1E as assessed using the information collected at screening
- Estimated glomerular filtration rate (eGFR) greater than or equal to (>=) 45 milliliters per minutes per 1.73 square meters (mL/min/1.73m^2) (based on the Chronic kidney disease- Epidemiology Collaboration [CKD-EPI 2021] eGFR equation) and is not anticipated by the participant's regular treating physician to have a sustained decline by greater than (>)10 percent (%) over the following 12 months
- Intolerant of tolvaptan treatment, unwilling to initiate tolvaptan treatment or ineligible for tolvaptan treatment for ADPKD
- A female participant is eligible to participate if she is not pregnant or breastfeeding and agrees to use birth control methods as discussed with the study doctor.
- Woman of childbearing potential (WOCBP) and Woman of non-childbearing potential (WONCBP) must have a negative highly sensitive pregnancy test prior to the Magnetic resonance imaging (MRI) scan being performed in the screening period of Part B
- A WOCBP and WONCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 24 hours before the first dose of study intervention For Part B (additional inclusion criteria for optional cohorts 4-5 only)
- eGFR >=30 mL/min/1.73m^2 (based on the CKD-EPI 2021 eGFR equation) and, in the clinical judgement of the investigator, the participant:
- has been clinically stable for the 24 months before screening; and,
- is not anticipated by the participant's regular treating physician to have a sustained decline in kidney function by >10% over the following 12 months; and,
- is not anticipated to need renal replacement therapy in the next 12 months; and,
- does not have any other clinical, biochemical or familial feature at screening that suggests a clinically significant decline in kidney function is anticipated in the next 12 months.
Who cannot join
- For Part A:
- History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drug; or interfering with the interpretation of data.
- History of malignancy of any type.
- History of kidney disease or kidney abnormalities or eGFR less than (<) 90 milliliters per minute per 1.73 square meters (mL/min/1.73m^2) (based on the chronic kidney disease- Epidemiology Collaboration [CKD-EPI] 2021 eGFR equation) at screening.
- Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements (including St John's Wort) within 7 days or 5 half-lives (whichever is longer) prior to the first dose of study drug and for the duration of study participation.
- QT interval corrected (QTc)>450 milliseconds (msec)
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- Participation in this study would result in loss of blood or blood products in excess of 500 mL within 56 days
- Current enrolment or past participation in an investigational clinical trial in which an investigational medicinal product was administered within the following time periods prior to the first dosing day of the current study: 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product, whichever is longer
- Exposure to more than 4 investigational medicinal products within 12 months prior to dosing
- Significant allergy to humanized monoclonal antibodies
- Clinically significant multiple or severe drug allergies, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear Immunoglobulin A (IgA) dermatosis, toxic epidermal necrolysis, and exfoliative dermatitis)
- Pregnant or lactating female
- Current or previous diagnosis of diabetes mellites (DM) (Type 1 or Type 2)
- Glycosylated hemoglobin (HbA1c) >=48 millimoles per mole (mmol/mol) (>=6.5%) at screening
- Bone fracture within 6 months prior to screening, or presence of a known unresolved or incompletely resolved fracture
- Positive pre-clinical study drug/alcohol screen, including tetrahydrocannabinol
- Positive Human Immunodeficiency Virus (HIV) antibody test
- Evidence of Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, as determined by local diagnostic procedures
- Evidence at screening of clinically significant hematological disorder (affecting hemoglobin, red blood cells [RBC], White blood cells [WBC] or platelets) or abnormal blood clotting parameters
- Regular use of recreational drugs, including substances containing tetrahydrocannabinol
- Participants who are unable to refrain from smoking, vaping or using other tobacco products during study visits or overnight stays
- Poor peripheral venous access by visual inspection (intravenous [IV] administration cohort(s) only)
- Average weekly intake of greater than (>) 14 United Kingdom (UK) units of alcohol. One UK unit is equivalent to 8 grams (g) of alcohol: a half-pint (approximately 240 mL) of beer, 1 glass (125 mL) of wine or 1 (25 mL) measure of spirits
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the investigator [or medical monitor], contraindicates participation in the study
- Use of any products intended to treat medical conditions that are not approved by the governing health authority in a given country or region (for example, herbal medicine, health supplements, traditional medicine, homeopathic remedies, etc.)
- Alanine transaminase (ALT) >1.5x Upper Limit of Normal (ULN)
- Total bilirubin >1.5xULN; Participants with Gilbert's syndrome can be included with total bilirubin <=3.0xULN as long as direct bilirubin is <=1.0xULN
- Current or chronic history of liver disease or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)
- Presence of hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) at screening or within 3 months prior to first dose of study intervention
- Positive hepatitis C antibody test result at screening or within 3 months prior to first dose of study intervention
- Positive hepatitis C Ribonucleic acid (RNA) test result at screening or within 3 months prior to first dose of study intervention
- Part B:
- Presence of active, clinically significant cardiovascular, respiratory, hepatic, gastrointestinal, endocrine (including, but not limited to, incompletely controlled secondary or tertiary hyperparathyroidism and clinically significant vitamin D deficiency), hematological (including, but not limited to, transfusion-dependent anemia), bone (including, but not limited to, osteoporosis), or neurological disorders, with the exception of ADPKD, capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drug; or interfering with the interpretation of data
- Clinically significant abnormal blood pressure (BP), that is, if the participant is not taking antihypertensive therapy: systolic BP >=160 millimeters of mercury (mmHg) and/or diastolic BP >=90mmHg; or If the participant is established on a stable regimen of antihypertensive drug(s): on-treatment systolic BP >=160 mmHg and/or diastolic BP >=90mmHg
- Lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of recurrence or metastatic disease for 3 years
- Breast cancer within the past 10 years
- Use of prescription and non-prescription drugs, including vitamins A, B, C, E, K, herbal and dietary supplements (including St John's Wort) within 7 days or 5 half-lives (whichever is longer) prior to the first dose of study drug and for the duration of study participation. Paracetamol, at doses of <=4 gram per 24 hour (g/24h) is permitted for use at any time during the study
- Contraindication to, or unwillingness to undergo, MRI scanning (e.g., presence of MRI-incompatible metal implant)
- Congenital absence of one kidney
- Kidney cyst interventions such as cyst aspiration or cyst fenestration within 12 weeks prior to screening and during the screening period, or such interventions planned or anticipated within the follow-up period
- Acute symptomatic kidney cyst hemorrhage or infection within 12 weeks prior to screening
- Evidence of current, chronic, or recurrent kidney or liver cyst infection
- Estimated proteinuria >1g/24 hour at screening and/or pre-dose (day -7 to day -1)
- Abnormal urinalysis suggestive of clinically significant glomerular disease or urinary tract infection
- Presence of known clinically significant renal or hepatic calculi, or symptoms thereof, at screening
- Treatment with tolvaptan within 6 months prior to screening
- Presence of an incompletely healed wound at screening and/or planned surgical procedure that would occur during study.
- Participation in this study would result in loss of blood or blood products in excess of 500 mL within 56 days
- Current enrolment or past participation in an investigational clinical trial in which an investigational medicinal product was administered within the following time periods prior to the first dosing day of the current study: 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product, whichever is longer
- Exposure to more than 4 investigational medicinal products within 12 months prior to dosing
- Significant allergy to humanized monoclonal antibodies
- Clinically significant multiple or severe drug allergies, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear Immunoglobulin A (IgA) dermatosis, toxic epidermal necrolysis, and exfoliative dermatitis)
- Pregnant or lactating female
- Current or previous diagnosis of diabetes mellites (DM) (Type 1 or Type 2)
- HbA1c >=48 millimoles per mole (mmol/mol) (>=6.5%) at screening
- Bone fracture within 6 months prior to screening, or presence of a known unresolved or incompletely resolved fracture
- Positive pre-clinical study drug/alcohol screen, including tetrahydrocannabinol
- Positive HIV antibody test
- Evidence of SARS-CoV-2 infection, as determined by local diagnostic procedures
- Evidence at screening of clinically significant hematological disorder (affecting hemoglobin, RBC, WBC or platelets) or abnormal blood clotting parameters
- Regular use of recreational drugs, including substances containing tetrahydrocannabinol
- Participants who are unable to refrain from smoking, vaping or using other tobacco products during study visits or overnight stays
- Poor peripheral venous access by visual inspection (IV administration cohort(s) only)
- Average weekly intake of greater than (>) 14 UK units of alcohol. One UK unit is equivalent to 8 grams of alcohol: a half-pint (approximately240 mL) of beer, 1 glass (125 mL) of wine or 1 (25 mL) measure of spirits
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the investigator [or medical monitor], contraindicates participation in the study
- Use of any products intended to treat medical conditions that are not approved by the governing health authority in a given country or region (for example, herbal medicine, health supplements, traditional medicine, homeopathic remedies, etc.)
- Alanine transaminase (ALT) >1.5x Upper Limit of Normal (ULN)
- Total bilirubin >1.5xULN; Participants with Gilbert's syndrome can be included with total bilirubin <=3.0xULN as long as direct bilirubin is <=1.0xULN
- Current or chronic history of liver disease or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)
- Presence of HBsAg and/or HBcAb at screening or within 3 months prior to first dose of study intervention
- Positive hepatitis C antibody test result at screening or within 3 months prior to first dose of study intervention
- Positive hepatitis C RNA test result at screening or within 3 months prior to first dose of study intervention
- QTc >450 milliseconds (msec) or QTc >480 msec for participants with bundle branch block
Trial locations and contacts
23 sites in 8 countries.
23 sites in 8 countries
Spain5 sites
GSK Investigational Site
Barcelona, 08003
GSK Investigational Site
Barcelona, 08025
GSK Investigational Site
Barcelona, 08035
GSK Investigational Site
Madrid, 28040
GSK Investigational Site
Madrid, 28041
United Kingdom4 sites
GSK Investigational Site
Cambridge, CB2 0GG
GSK Investigational Site
Exeter, EX2 5DW
GSK Investigational Site
London, NW3 2QG
GSK Investigational Site
Newcastle upon Tyne, NE1 4LP
Belgium3 sites
GSK Investigational Site
Brussels, 1200
GSK Investigational Site
Ghent, 9000
GSK Investigational Site
Leuven, 3000
Canada3 sites
GSK Investigational Site
London, Ontario, N6A 5A5
GSK Investigational Site
Montreal, Quebec, H4J 1C5
GSK Investigational Site
Toronto, Ontario, M5G 2C4
Netherlands3 sites
GSK Investigational Site
Groningen, 9713 GZ
GSK Investigational Site
Leiden, 2333 ZA
GSK Investigational Site
Rotterdam, 3015 GD
France2 sites
GSK Investigational Site
Brest, 29200
GSK Investigational Site
Paris, 75015
Germany2 sites
GSK Investigational Site
Cologne, North Rhine-Westphalia, 50937
GSK Investigational Site
Lübeck, Schleswig-Holstein, 23538
United States1 site
GSK Investigational Site
Rochester, Minnesota, 55905
About this study
Official title: 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
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).
- Conditions
- Kidney Disease
- Being tested
- GSK4771261, Placebo matching GSK4771261
- Keywords
- Phase 1, GSK4771261, Autosomal dominant polycystic kidney disease (ADPKD), Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity
- Medical subject headings
- Kidney Diseases, Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GSK4771261Drug
GSK4771261 will be administered.
Placebo matching GSK4771261Drug
Placebo matching GSK4771261 will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Cohort 1: Participants receiving GSK4771261 Dose level 1 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 1. Receives: Drug: GSK4771261 |
| Part A: Cohort 2 : Participants receiving GSK4771261 Dose level 2 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 2. Receives: Drug: GSK4771261 |
| Part A: Cohort 3: Participants receiving GSK4771261 Dose level 3 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 3. Receives: Drug: GSK4771261 |
| Part A: Cohort 4: Participants receiving GSK4771261 Dose level 4 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 4. Receives: Drug: GSK4771261 |
| Part A: Cohort 5: Participants receiving GSK4771261 Dose level 5 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 5. Receives: Drug: GSK4771261 |
| Part A: Cohort 6: Participants receiving GSK4771261 Dose level 6 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 6. Receives: Drug: GSK4771261 |
| Part A: Cohort 7 (optional): Participants receiving GSK4771261 Dose level 7 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 7 as the recommendation of Dose escalation committee (DEC). Receives: Drug: GSK4771261 |
| Part A: Cohort 8 (optional): Participants receiving GSK4771261 Dose level 8 | Experimental | Healthy participants will be randomized to receive GSK4771261 Dose level 8, as per the recommendation from DEC. Receives: Drug: GSK4771261 |
| Part B: Cohort 1: Participants receiving GSK4771261 Dose level 9 | Experimental | Participant with Autosomal dominant polycystic kidney disease (ADPKD) will be randomized to receive GSK4771261 Dose level 9. Receives: Drug: GSK4771261 |
| Part B: Cohort 2: Participants receiving GSK4771261 Dose level 10 | Experimental | Participant with ADPKD will be randomized to receive GSK4771261 Dose level 10. Receives: Drug: GSK4771261 |
| Part B: Cohort 3: Participants receiving GSK4771261 Dose level 11 | Experimental | Participant with ADPKD will be randomized to receive GSK4771261 Dose level11. Receives: Drug: GSK4771261 |
| Part B: Cohort 4 (optional): Participants receiving GSK4771261 Dose level 12 | Experimental | Participant with ADPKD will be randomized to receive GSK4771261 Dose level 12 as the recommendation from DEC. Receives: Drug: GSK4771261 |
| Part B: Cohort 5 (optional): Participants receiving GSK4771261 Dose level 13 | Experimental | Participant with ADPKD will be randomized to receive GSK4771261 Dose level 13, as per the recommendation from DEC. Receives: Drug: GSK4771261 |
| Part A: Participants receiving Placebo matching GSK4771261 | Placebo comparator | Participants will receive placebo matching GSK4771261 Receives: Drug: Placebo matching GSK4771261 |
| Part B: Participants receiving placebo matching GSK4771261 | Placebo comparator | Participants will receive placebo matching GSK4771261 Receives: Drug: Placebo matching GSK4771261 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Number of Participants with Adverse events (AEs)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Measured: From Baseline (Day 1) up to Day 182 (End of study [EoS])
Part B: Number of Participants with AEs
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part A: Number of Participants with Serious Adverse Events (SAEs)
An SAE is defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: results in death; is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect in the offspring of a study participant; abnormal pregnancy outcome (for example, spontaneous abortion, fetal death, stillbirth, congenital anomalies, ectopic pregnancy); is a suspected transmission of any infectious agent via an authorized medicinal product; or other situations.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with SAEs
An SAE is defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: results in death; is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect in the offspring of a study participant; abnormal pregnancy outcome (for example, spontaneous abortion, fetal death, stillbirth, congenital anomalies, ectopic pregnancy); is a suspected transmission of any infectious agent via an authorized medicinal product; or other situations
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part A: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Number of participants with clinically significant changes in blood laboratory values will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Number of participants with clinically significant changes in blood laboratory values will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part A: Number of Participants with Clinically Significant Changes in Urine Laboratory Values
Number of participants with clinically significant changes in Urine laboratory values will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Clinically Significant Changes in Urine Laboratory Values
Number of participants with clinically significant changes in Urine laboratory values will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part A: Number of Participants with Clinically Significant Changes in Vital Signs
Number of participants with clinically significant changes in vital signs (will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Clinically Significant Changes in Vital Signs
Number of participants with clinically significant changes in vital signs will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part A: Number of Participants with Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Number of participants with clinically significant changes in 12-lead ECG parameters will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Clinically Significant Changes in 12-lead ECG
Number of participants with clinically significant changes in 12-lead ECG parameters will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Secondary outcomes (8)
Part A: Number of Participants with Anti-drug Antibodies for GSK4771261
Number of participants with Anti-drug antibodies for GSK4771261 will be evaluated.
Measured: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Anti-drug Antibodies for GSK4771261
Number of participants with Anti-drug antibodies for GSK4771261 will be evaluated.
Measured: 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
Blood samples were collected to evaluate pharmacokinetics of GSK4771261.
Measured: Up to Day 182
Part A: Area under the concentration-time curve to infinity AUC (0-inf) of GSK4771261
Blood samples were collected to evaluate pharmacokinetics of GSK4771261.
Measured: Up to Day 182
Part B: AUC (0-tau) of GSK4771261
Blood samples were collected to evaluate pharmacokinetics of GSK4771261.
Measured: Up to Day 182
Part B: AUC (0-inf) of GSK4771261
Blood samples were collected to evaluate pharmacokinetics of GSK4771261.
Measured: Up to Day 182
Part A: Maximum Plasma Concentration (Cmax) of GSK4771261
Blood samples were collected to evaluate of GSK4771261.
Measured: Up to Day 182
Part B: Cmax of GSK4771261
Blood samples were collected to evaluate of GSK4771261.
Measured: Up to Day 182
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2024
- Study start11 Dec 2024
- Primary completion23 Jul 2027 (estimated)
- Study completion23 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Kidney DiseaseStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- This is sequential double-blind study.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 86 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06734234
- Sponsor's protocol ID
- 221362
- Other IDs
- 2024-516095-15
- Status last verified
- Jul 2026
- First submitted
- 11 Dec 2024
- Last updated
- 30 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: GSK will assess requests from qualified researchers for anonymized individual patient-level data and related study documents. Data sharing is subject to certain criteria, conditions, and exceptions. For further information, refer to https://www.gsk-studyregister.com/About\_GSK\_Patient\_Level\_Data\_Sharing\_Final\_13July2023.pdf
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06734234
- Acronym / sponsor ID
- 221362
- Conditions
- Kidney Disease
- Keywords
- Phase 1, GSK4771261, Autosomal dominant polycystic kidney disease (ADPKD), Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 86 participants (estimated)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- 11 Dec 2024 (actual)
- Primary completion
- 23 Jul 2027 (estimated)
- Study completion
- 23 Jul 2027 (estimated)
- First posted
- 16 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 15
- Model description
- This is sequential double-blind study.
| Arm | Type | Receives |
|---|---|---|
| Part A: Cohort 1: Participants receiving GSK4771261 Dose level 1 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 1. |
| Part A: Cohort 2 : Participants receiving GSK4771261 Dose level 2 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 2. |
| Part A: Cohort 3: Participants receiving GSK4771261 Dose level 3 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 3. |
| Part A: Cohort 4: Participants receiving GSK4771261 Dose level 4 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 4. |
| Part A: Cohort 5: Participants receiving GSK4771261 Dose level 5 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 5. |
| Part A: Cohort 6: Participants receiving GSK4771261 Dose level 6 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 6. |
| Part A: Cohort 7 (optional): Participants receiving GSK4771261 Dose level 7 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 7 as the recommendation of Dose escalation committee (DEC). |
| Part A: Cohort 8 (optional): Participants receiving GSK4771261 Dose level 8 | Experimental | Drug: GSK4771261Healthy participants will be randomized to receive GSK4771261 Dose level 8, as per the recommendation from DEC. |
| Part B: Cohort 1: Participants receiving GSK4771261 Dose level 9 | Experimental | Drug: GSK4771261Participant with Autosomal dominant polycystic kidney disease (ADPKD) will be randomized to receive GSK4771261 Dose level 9. |
| Part B: Cohort 2: Participants receiving GSK4771261 Dose level 10 | Experimental | Drug: GSK4771261Participant with ADPKD will be randomized to receive GSK4771261 Dose level 10. |
| Part B: Cohort 3: Participants receiving GSK4771261 Dose level 11 | Experimental | Drug: GSK4771261Participant with ADPKD will be randomized to receive GSK4771261 Dose level11. |
| Part B: Cohort 4 (optional): Participants receiving GSK4771261 Dose level 12 | Experimental | Drug: GSK4771261Participant with ADPKD will be randomized to receive GSK4771261 Dose level 12 as the recommendation from DEC. |
| Part B: Cohort 5 (optional): Participants receiving GSK4771261 Dose level 13 | Experimental | Drug: GSK4771261Participant with ADPKD will be randomized to receive GSK4771261 Dose level 13, as per the recommendation from DEC. |
| Part A: Participants receiving Placebo matching GSK4771261 | Placebo comparator | Drug: Placebo matching GSK4771261Participants will receive placebo matching GSK4771261 |
| Part B: Participants receiving placebo matching GSK4771261 | Placebo comparator | Drug: Placebo matching GSK4771261Participants will receive placebo matching GSK4771261 |
Who may qualify
- Ages
- 25 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- 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
Key exclusion criteria (73 listed)
- History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drug; or interfering with the interpretation of data.
- History of malignancy of any type.
- History of kidney disease or kidney abnormalities or eGFR less than (<) 90 milliliters per minute per 1.73 square meters (mL/min/1.73m^2) (based on the chronic kidney disease- Epidemiology Collaboration [CKD-EPI] 2021 eGFR equation) at screening.
- Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements (including St John's Wort) within 7 days or 5 half-lives (whichever is longer) prior to the first dose of study drug and for the duration of study participation.
- QT interval corrected (QTc)>450 milliseconds (msec)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GSK4771261GSK4771261 will be administered. | Drug | Part A: Cohort 1: Participants receiving GSK4771261 Dose level 1, Part A: Cohort 2 : Participants receiving GSK4771261 Dose level 2, Part A: Cohort 3: Participants receiving GSK4771261 Dose level 3, Part A: Cohort 4: Participants receiving GSK4771261 Dose level 4, Part A: Cohort 5: Participants receiving GSK4771261 Dose level 5, Part A: Cohort 6: Participants receiving GSK4771261 Dose level 6, Part A: Cohort 7 (optional): Participants receiving GSK4771261 Dose level 7, Part A: Cohort 8 (optional): Participants receiving GSK4771261 Dose level 8, Part B: Cohort 1: Participants receiving GSK4771261 Dose level 9, Part B: Cohort 2: Participants receiving GSK4771261 Dose level 10, Part B: Cohort 3: Participants receiving GSK4771261 Dose level 11, Part B: Cohort 4 (optional): Participants receiving GSK4771261 Dose level 12, Part B: Cohort 5 (optional): Participants receiving GSK4771261 Dose level 13 |
| Placebo matching GSK4771261Placebo matching GSK4771261 will be administered. | Drug | Part A: Participants receiving Placebo matching GSK4771261, Part B: Participants receiving placebo matching GSK4771261 |
Primary outcome
Part A: Number of Participants with Adverse events (AEs)
Time frame: From Baseline (Day 1) up to Day 182 (End of study [EoS])
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Part B: Number of Participants with AEs
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Part A: Number of Participants with Serious Adverse Events (SAEs)
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
An SAE is defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: results in death; is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect in the offspring of a study participant; abnormal pregnancy outcome (for example, spontaneous abortion, fetal death, stillbirth, congenital anomalies, ectopic pregnancy); is a suspected transmission of any infectious agent via an authorized medicinal product; or other situations.
Part B: Number of Participants with SAEs
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
An SAE is defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: results in death; is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect in the offspring of a study participant; abnormal pregnancy outcome (for example, spontaneous abortion, fetal death, stillbirth, congenital anomalies, ectopic pregnancy); is a suspected transmission of any infectious agent via an authorized medicinal product; or other situations
Part A: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in blood laboratory values will be evaluated.
Part B: Number of Participants with Clinically Significant Changes in Blood Laboratory Values
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in blood laboratory values will be evaluated.
Part A: Number of Participants with Clinically Significant Changes in Urine Laboratory Values
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in Urine laboratory values will be evaluated.
Part B: Number of Participants with Clinically Significant Changes in Urine Laboratory Values
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in Urine laboratory values will be evaluated.
Part A: Number of Participants with Clinically Significant Changes in Vital Signs
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in vital signs (will be evaluated.
Part B: Number of Participants with Clinically Significant Changes in Vital Signs
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in vital signs will be evaluated.
Part A: Number of Participants with Clinically Significant Changes in 12-lead Electrocardiogram (ECG)
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in 12-lead ECG parameters will be evaluated.
Part B: Number of Participants with Clinically Significant Changes in 12-lead ECG
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Number of participants with clinically significant changes in 12-lead ECG parameters will be evaluated.
8 secondary outcomes
Part A: Number of Participants with Anti-drug Antibodies for GSK4771261
Time frame: From Baseline (Day 1) up to Day 182 (EoS)
Part B: Number of Participants with Anti-drug Antibodies for GSK4771261
Time frame: 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
Time frame: Up to Day 182
Part A: Area under the concentration-time curve to infinity AUC (0-inf) of GSK4771261
Time frame: Up to Day 182
Part B: AUC (0-tau) of GSK4771261
Time frame: Up to Day 182
Part B: AUC (0-inf) of GSK4771261
Time frame: Up to Day 182
Part A: Maximum Plasma Concentration (Cmax) of GSK4771261
Time frame: Up to Day 182
Part B: Cmax of GSK4771261
Time frame: Up to Day 182
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2026.
- Individual participant dataSharing planned: Yes. GSK will assess requests from qualified researchers for anonymized individual patient-level data and related study documents. Data sharing is subject to certain criteria, conditions, and exceptions. For further information, refer to https://www.gsk-studyregister.com/About_GSK_Patient_Level_Data_Sharing_Final_13July2023.pdf
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GSK Clinical Trials study director
GlaxoSmithKline
23 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GSK4771261 in people with Kidney Disease, run by GlaxoSmithKline at 23 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 73 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Spain, United Kingdom, Belgium, Canada, Netherlands and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Number of Participants with Adverse events (AEs) (time frame: From Baseline (Day 1) up to Day 182 (End of study [EoS])). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2026.