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Hematologic DiseasesPhase 1NCT05660265

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

A Phase 1 study testing GSK4172239D in people with Hematologic Diseases and Anaemia, Sickle Cell, run by GlaxoSmithKline at 6 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 18 to 50 years
Conditions the study is for
Hematologic Diseases, Anaemia, Sickle Cell
What is being tested?
GSK4172239D
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
11 participants
Where
6 sitesUnited States
Study dates
26 Aug 2023to 22 Sep 2025
Phase
Phase 1
Are there results?
None posted in the registry

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 18 to 50 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • 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.

Who cannot join

  • 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%).
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  • 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.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • GSK Investigational Site

    Atlanta, Georgia, 30315

  • GSK Investigational Site

    Columbus, Georgia, 31904

  • GSK Investigational Site

    Las Vegas, Nevada, 89113

  • GSK Investigational Site

    Miami, Florida, 33155

  • GSK Investigational Site

    Riverdale, Georgia, 30274

  • GSK Investigational Site

    Tamarac, Florida, 33321

Other trials in United States

About this study

Official title: 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.

Being tested
GSK4172239D, Placebo
Keywords
Hematologic diseases, Sickle cell disease, First time in human study, 218471
Medical subject headings
Hematologic Diseases, Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GSK4172239DDrug

    Different strength of GSK4172239D will be administered in different cohorts.

  • PlaceboOther

    Matching placebo will be administered.

Study groups in NCT05660265
Study groupRoleWhat they receive
Cohort 1ExperimentalParticipants in this arm will receive either single dose of GSK4172239D (Dose 1) or matching placebo.
Receives: Drug: GSK4172239D; Other: Placebo
Cohort 2ExperimentalParticipants in this arm will receive either single dose of GSK4172239D (Dose 2) or matching placebo.
Receives: Drug: GSK4172239D; Other: Placebo
Cohort 3ExperimentalParticipants in this arm will receive either single dose of GSK4172239D (Dose 3) or matching placebo.
Receives: Drug: GSK4172239D; Other: Placebo
Cohort 4ExperimentalParticipants in this arm will receive either single dose of GSK4172239D (Dose 4) or matching placebo.
Receives: Drug: GSK4172239D; Other: Placebo
Cohort 5ExperimentalParticipants in this arm will receive either single dose of GSK4172239D (Dose 5) or matching placebo.
Receives: Drug: GSK4172239D; Other: Placebo
Food effect cohortExperimentalOne selected cohort will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions.
Receives: Drug: GSK4172239D; Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Area under curve zero to time infinity (AUC 0-inf) for GSK4106401 after a single oral dose of GSK4172239D

    Measured: Up to Day 3

  2. Maximum observed plasma concentration (Cmax) for GSK4106401 after a single oral dose of GSK4172239D

    Measured: Up to Day 3

  3. Time to Cmax (Tmax) for GSK4106401 after a single oral dose of GSK4172239D

    Measured: Up to Day 3

  4. Half-life (t1/2) for GSK4106401 after a single oral dose of GSK4172239D

    Measured: Up to Day 3

  5. Ratio between the fed and fasted conditions for AUC (0-inf)

    Measured: Up to Day 3

  6. Ratio between the fed and fasted conditions for Cmax

    Measured: Up to Day 3

Secondary outcomes (10)

  1. Number of participants with clinically significant changes from baseline in white blood cell (WBC)

    Measured: Baseline and up to Day 7

  2. Number of participants with clinically significant changes from baseline in hemoglobin

    Measured: Baseline and up to Day 7

  3. Number of participants with clinically significant changes from baseline in platelets count

    Measured: Baseline and up to Day 7

  4. Number of participants with clinically significant changes from baseline in neutrophil count

    Measured: Baseline and up to Day 7

  5. Number of participants with clinically significant changes from baseline in alanine transaminase (ALT)

    Measured: Baseline and up to Day 7

  6. Number of participants with clinically significant changes from baseline in aspartate transaminase (AST)

    Measured: Baseline and up to Day 7

  7. Number of participants with clinically significant changes from baseline in bilirubin

    Measured: Baseline and up to Day 7

  8. Number of participants with adverse event (AE) and serious adverse event (SAE)

    Measured: Up to Day 7

  9. Number of participants with clinically significant change from baseline in 12 lead electrocardiograms (ECG)

    Measured: Baseline and up to Day 7

  10. Number of participants with clinically significant change from baseline in vital signs

    Measured: Baseline and up to Day 7

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Dec 2022
  2. Study start26 Aug 2023
  3. Primary completion22 Sep 2025
  4. Study completion22 Sep 2025

Why it stopped: Sponsor decision to terminate the study due to strategic business consideration and not related to safety.

Resources

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
Parallel groups
Model description
This is a placebo controlled multi-center study.
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05660265
Sponsor's protocol ID
218471
Status last verified
Jan 2026
First submitted
13 Dec 2022
Last updated
22 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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/

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing GSK4172239D in people with Hematologic Diseases and Anaemia, Sickle Cell, run by GlaxoSmithKline at 6 sites in United States.

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 6 inclusion and 15 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: stopped early, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. 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. 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?

The study was stopped early (reason given: Sponsor decision to terminate the study due to strategic business consideration and not related to safety.). Results are not available in the source record.

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?

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). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05660265.

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 22 Jan 2026.

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