Hibiscus2 (Sickle Cell Disease)

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.

Who Can Participate?

Eligibility

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.

Age Range

12-17

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

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.

Locations

Other

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

No

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

Jennifer
Rothman

Protocol Number

PRO00116729

NCT ID

NCT06612268

Phase

III

Enrollment Status

Open to Enrollment
ClinicalTrials.gov