GSK4172239D for Sickle Cell Anemia

Recruiting at 10 trial locations

Overseen By EU GSK Clinical Trials Call Center

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: GlaxoSmithKline

Must not be taking: Hydroxyurea, Decitabine, St John's Wort

Disqualifiers: Cardiovascular, Hypertension, Heart disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, GSK4172239D, for people with sickle cell anemia. The main goal is to assess its safety and how the body processes it. Participants will receive either the treatment or a placebo (a harmless pill with no effect) in varying doses. The trial seeks individuals with sickle cell anemia who haven't found success with other treatments. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

Yes, you will need to stop taking most prescription and non-prescription drugs, including vitamins and supplements, at least 7 days before the study starts. However, you may take acetaminophen up to 48 hours before the first dose, and opioid or benzodiazepine use as directed by a physician is allowed.

Is there any evidence suggesting that GSK4172239D is likely to be safe for humans?

Researchers are investigating the safety of GSK4172239D for people with sickle cell disease. This marks the first human trial of the drug, so direct information on its tolerance is not yet available. However, such studies typically proceed with caution, beginning with small doses and closely monitoring participants for side effects. This careful approach helps determine the safest dose before expanding to more participants or larger doses. If the drug has been used in other trials or for other conditions, it might offer some insights into its safety. However, detailed safety information for GSK4172239D will emerge from this current study. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sickle cell anemia?

Researchers are excited about GSK4172239D for sickle cell anemia because it offers a potentially new approach to treating the disease. Unlike standard treatments, which often focus on managing symptoms or preventing complications, GSK4172239D may directly target the underlying mechanisms of sickle cell anemia. This could lead to more effective management of the disease. Its unique mechanism of action, possibly at a molecular level, might offer benefits that current therapies don't provide, sparking hope for better patient outcomes.

What evidence suggests that GSK4172239D might be an effective treatment for sickle cell anemia?

Research has shown that GSK4172239D could be a promising treatment for sickle cell disease (SCD). This trial will evaluate different doses of GSK4172239D, with participants in various arms receiving either a single dose of the drug or a matching placebo. The drug converts into an active form in the body and targets specific cells to potentially ease symptoms. Although direct evidence of its effectiveness in humans is not yet available, its mechanism suggests it might help with SCD-related issues. Current understanding is based on its cellular interactions, aiming to improve outcomes for people with SCD. As trials progress, researchers will gather more solid evidence about its efficacy. ^{1 2 4 5 6}

Who Is on the Research Team?

GSK Clinical Trials

Principal Investigator

GlaxoSmithKline

Are You a Good Fit for This Trial?

Adults aged 18-50 with Sickle Cell Disease (SCD), not on gamma-globin increasing meds, weighing over 50 kg, and able to consent. Men must practice abstinence or use contraception; women must be non-childbearing. Excludes those with certain blood counts, drug abuse history, significant heart disease, organ disorders affecting drug metabolism, abnormal blood pressure or kidney function, recent transfusions or certain medications.

Inclusion Criteria

I have SCD and am not on medication that increases fetal hemoglobin.

I have sickle cell disease and previous treatments didn't work for me.

My weight is over 50 kilograms.

Exclusion Criteria

I haven't taken any drugs or supplements that could affect the study in the last 7 to 14 days, except for low-dose acetaminophen.

Absolute neutrophil count less than 1,500 / microlitre (μL)

I have not used hydroxyurea or decitabine in the last 9 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of GSK4172239D or placebo, with dose escalation and a food effect cohort

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and pharmacokinetics after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• GSK4172239D

Trial Overview The trial is testing GSK4172239D's safety and how the body processes it compared to a placebo in people with SCD. It involves three phases: screening, treatment where participants are randomly given either the study drug or placebo in varying doses including under fed conditions for some after a break period.

How Is the Trial Designed?

Group I: Food effect cohort Experimental Treatment 2 Interventions

One selected cohort will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions.

Group II: Cohort 5 Experimental Treatment 2 Interventions

Participants in this arm will receive either single dose of GSK4172239D (Dose 5) or matching placebo.

Group III: Cohort 4 Experimental Treatment 2 Interventions

Participants in this arm will receive either single dose of GSK4172239D (Dose 4) or matching placebo.

Group IV: Cohort 3 Experimental Treatment 2 Interventions

Participants in this arm will receive either single dose of GSK4172239D (Dose 3) or matching placebo.

Group V: Cohort 2 Experimental Treatment 2 Interventions

Participants in this arm will receive either single dose of GSK4172239D (Dose 2) or matching placebo.

Group VI: Cohort 1 Experimental Treatment 2 Interventions

Participants in this arm will receive either single dose of GSK4172239D (Dose 1) or matching placebo.

Who Is Running the Clinical Trial?

GlaxoSmithKline

Lead Sponsor

Trials

4,834

Recruited

8,389,000+

Headquarters

London, UK

Known For

Vaccines & Medicines

Top Products

**Advair (salmeterol, fluticasone propionate)**, **Shingrix (shingles vaccine)**, **Augmentin (amoxicillin/clavulanate potassium)**, **Ventolin (salbutamol sulfate)

Dame Emma Walmsley

GlaxoSmithKline

Chief Executive Officer since 2017

MA in Classics and Modern Languages from Oxford University

Dr. Hal Barron

GlaxoSmithKline

Chief Medical Officer since 2018

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05660265

NCT05660265 | A Study to Investigate the Safety, ... The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D. The study will be composed of 3 periods for all ...

2. ctsearchsupport.org ctsearchsupport.org/clinical-trials/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 drug, GSK4172239D, to treat sickle cell disease (SCD) GSK4172239D is a drug that targets DNMT1 in certain cells. Everyone in this trial will get standard drugs to treat pain.

3. gsk.com gsk.com/media/ofspbxv2/q2-2025-pipeline-assets-and-clinical-trials-report.pdf

Pipeline assets and clinical trials appendix - Q2 2025 GSK4172239 (Sickle cell disease). NCT05660265. Phase. I. Patient. Participants ... A multicentre, randomised, open-label, trial comparing the efficacy, safety, PK ...

4. gsk.com gsk.com/en-gb/innovation/pipeline/

Pipeline At GSK, our portfolio and pipeline is focused in developing medicines and vaccines in four core therapeutic areas.

5. withpower.com withpower.com/trial/phase-1-anemia-sickle-cell-11-2022-ddd6c

GSK4172239D for Sickle Cell Anemia This trial is testing a new drug called GSK4172239D in people with sickle cell disease. The drug changes into an active form inside the body to help treat ...

6. synapse.patsnap.com synapse.patsnap.com/drug/36e47efff9514947afa9bbd927168a1d

GSK4172239D - Drug Targets, Indications, Patents Phase I Study in Sickle Cell Disease Participants, to Evaluate the Safety, Tolerability, and Pharmacokinetics of GSK4172239D