Clinical Trial 52779

Miami, FL 33147


Summary:

This will be a first time in human study in sickle cell diseases (SCD) participants. The 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).

This study will be a single dose, dose-escalation study.


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 and agree to remain abstinent. OR agree to use a male condom with female partner
  • For female participants: Female participants are eligible to participate if they are a woman of non-childbearing potential (WONCBP).
  • Capable of giving informed consent.


Qualified Participants May Receive:

Great care and close monitorting 


Clinical trials are medical research studies designed to test the safety and/or effectiveness of new investigational drugs, devices, or treatments in humans. These studies are conducted worldwide for a range of conditions and illnesses. Learn more about clinical research and participating in a study at About Clinical Trials.