Hibiscus2 (Sickle Cell Disease) - Clinical Trial

What is the Purpose of this Study?

We are doing this study to find out if an experimental drug called etavopivat (the study drug) is a safe and effective option for children and adolescents who have SCD.

What is the Condition Being Studied?

Enfermedad de células falciformes (SCD)

Who Can Participate in the Study?

Children ages 12-17 who:

  • Are diagnosed with SCD
  • Have had at least 2, but more than 15, documented vaso-occlusive crises (pain crises)

For more information, contact the study team at morgan.low@duke.edu.

Grupo etario
Adults, Children

What is Involved?

If you and your child choose to take part in this study, they will get a random assignment (like a coin flip) to either:

  • Take a 400mg dose of the study drug; OR
  • Take a placebo (inactive substance with no drug in it)

Your child will take the study drug or placebo for 1 year. After this year is finished, you and they will have the option to continue on to Part 2 of the study. In Part 2, everyone will receive a 400mg dose of the study drug regardless of whether or not they took the study drug or placebo for the first year. Part 2 will also last for 1 year.

Study Details

Full Title
A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease
Principal Investigator
Especialista en hematología-oncología pediátrica
Protocol Number
IRB: PRO00116729
NCT: NCT06612268
Phase
Phase III
ClinicalTrials.gov
Estado de inscripción
Abierto para inscripción