A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

January 20, 2026 updated by: GlaxoSmithKline

A Randomized, Placebo-controlled, Double-Blind (Sponsor Unblind), Parallel Group, Single Dose, Dose Escalation Phase I Study in Sickle Cell Disease Participants, to Evaluate the Safety, Tolerability, and Pharmacokinetics of GSK4172239D

This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D.

The study will be composed of 3 periods for all participants (Screening, Treatment, and Follow up). Participants will be screened and, prior to first dose on Day 1, will be randomized to receive either GSK4172239D or placebo.

GSK4172239D is a prodrug that is converted in vivo into GSK4106401. This study will be a single dose, dose-escalation study. The initial dosing for all cohorts will be staggered so that 2 participants will be dosed as sentinel participants. Provided there are no safety concerns in 48 hours (h), the remaining 6 participants scheduled for the cohort may be dosed. One selected cohort of participants will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions after a washout period of a minimum of 20 days or 5 half-lives, whichever is longer, designated as the Food Effect Cohort.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
11

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33155
        • GSK Investigational Site
      • Tamarac, Florida, United States, 33321
        • GSK Investigational Site
    • Georgia
      • Atlanta, Georgia, United States, 30315
        • GSK Investigational Site
      • Columbus, Georgia, United States, 31904
        • GSK Investigational Site
      • Riverdale, Georgia, United States, 30274
        • GSK Investigational Site
    • Nevada
      • Las Vegas, Nevada, United States, 89113
        • GSK Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 50 years (Adult)

Accepts Healthy Volunteers

No

Description

Inclusion criteria:

  • Participants diagnosed with SCD not taking medication which increases gamma-globin (fetal hemoglobin).
  • Participants with SCD who have failed or not tolerated one or more approved therapies for SCD
  • Body weight greater than (>) 50 kilogram (kg).
  • For male participants: Refrain from donating sperm plus either be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent. OR agree to use a male condom with female partner. Agree to use an additional highly effective contraceptive method with a failure rate of less than (<) 1% per year when having sexual intercourse with a woman of childbearing potential who is not currently pregnant
  • For female participants: Female participants are eligible to participate if they are a woman of non-childbearing potential (WONCBP).
  • Capable of giving informed consent.

Exclusion Criteria:

  • Presence of active, clinically significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, 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.
  • Clinically significant abnormal blood pressure and/or history of hypertension as determined by the investigator.
  • History of clinically significant heart disease as determined by the investigator.
  • Estimated glomerular filtration rate (eGFR) < 60 ml/min/1.73m^2
  • ALT > 3x upper limit of normal (ULN).
  • Bilirubin > 5x ULN (isolated bilirubin > 5x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
  • Hemoglobin < 6 gram/decalitre (g/dL).
  • Absolute neutrophil count <1,500 / microlitre (μL).
  • Platelet count <75,000 /μL or >750,000 /μL.
  • Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements (including St John's Wort) within 7 days (or 14 days if the drug is a potential enzyme inducer) or 5 t1/2 (whichever is longer) prior to the first dose of study drug, unless in the opinion of the Investigator and GSK Medical Monitor the medication will not interfere with the study procedures or compromise participant safety. By exception, participant may take acetaminophen (less than or equal to [≤] 2 g/day) up to 48h prior to the first dose of study drug.
  • Use of hydroxyurea or decitabine within 9 weeks prior to baseline through follow-up.
  • Blood transfusion within 3 months prior to baseline through follow-up.
  • Current enrollment or past participation within the last 30 days before signing of consent in this or any other clinical study involving an investigational study drug or any other type of medical research.
  • Positive pre-study drug/alcohol screen. By exception, opioid use for pain or benzodiazepine use for anxiety as directed by a physician is permitted.
  • Regular use of known drugs of abuse, except for use directed by a physician. By exception, opioid use for pain or benzodiazepine use for anxiety is permitted.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort 1
Participants in this arm will receive either single dose of GSK4172239D (Dose 1) or matching placebo.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.
Experimental: Cohort 2
Participants in this arm will receive either single dose of GSK4172239D (Dose 2) or matching placebo.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.
Experimental: Cohort 3
Participants in this arm will receive either single dose of GSK4172239D (Dose 3) or matching placebo.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.
Experimental: Cohort 4
Participants in this arm will receive either single dose of GSK4172239D (Dose 4) or matching placebo.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.
Experimental: Cohort 5
Participants in this arm will receive either single dose of GSK4172239D (Dose 5) or matching placebo.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.
Experimental: Food effect cohort
One selected cohort will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions.
Other: Placebo
Matching placebo will be administered.
Drug: GSK4172239D
Different strength of GSK4172239D will be administered in different cohorts.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Area under curve zero to time infinity (AUC 0-inf) for GSK4106401 after a single oral dose of GSK4172239D
Time Frame: Up to Day 3
Up to Day 3
Maximum observed plasma concentration (Cmax) for GSK4106401 after a single oral dose of GSK4172239D
Time Frame: Up to Day 3
Up to Day 3
Time to Cmax (Tmax) for GSK4106401 after a single oral dose of GSK4172239D
Time Frame: Up to Day 3
Up to Day 3
Half-life (t1/2) for GSK4106401 after a single oral dose of GSK4172239D
Time Frame: Up to Day 3
Up to Day 3
Ratio between the fed and fasted conditions for AUC (0-inf)
Time Frame: Up to Day 3
Up to Day 3
Ratio between the fed and fasted conditions for Cmax
Time Frame: Up to Day 3
Up to Day 3

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Number of participants with clinically significant changes from baseline in white blood cell (WBC)
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in hemoglobin
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in platelets count
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in neutrophil count
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in alanine transaminase (ALT)
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in aspartate transaminase (AST)
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant changes from baseline in bilirubin
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with adverse event (AE) and serious adverse event (SAE)
Time Frame: Up to Day 7
Up to Day 7
Number of participants with clinically significant change from baseline in 12 lead electrocardiograms (ECG)
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7
Number of participants with clinically significant change from baseline in vital signs
Time Frame: Baseline and up to Day 7
Baseline and up to Day 7

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
GlaxoSmithKline

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: GSK Clinical Trials, GlaxoSmithKline

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 26, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 22, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 22, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 13, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 13, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 21, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 22, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 20, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 218471

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to anonymized individual patient-level data (IPD) and related study documents of the eligible studies via the Data Sharing Portal. Details on GSK's data sharing criteria can be found at: https://www.gsk.com/en-gb/innovation/trials/data-transparency/

IPD Sharing Time Frame

Anonymized IPD will be made available within 6 months of publication of primary, key secondary and safety results for studies in product with approved indication(s) or terminated asset(s) across all indications.

IPD Sharing Access Criteria

Anonymized IPD is shared with researchers whose proposals are approved by an Independent Review Panel and after a Data Sharing Agreement is in place. Access is provided for an initial period of 12 months but an extension may be granted, when justified, for up to 6 months.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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