GSK4172239D for Sickle Cell Anemia

Recruiting at11 trial locations

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: GlaxoSmithKline

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

Disqualifiers: Cardiovascular, Hypertension, Heart disease, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

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 the condition. The study aims to see if the drug is safe and how it behaves in the body.

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.

Research Team

GSK Clinical Trials

Principal Investigator

GlaxoSmithKline

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

• GSK4172239D (Prodrug)

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.

Participant Groups

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

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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