HIBISCUS Study 4202-HEM-301 (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 managing pain flares from sickle cell disease.

Who Can Participate?

Eligibility

Children and adults ages 12-65 who:
  • Are diagnosed with sickle cell disease
  • Have had at least 2 episodes of vaso-occlusive (pain) crisis in the past year
  • Have not changed their dose of hydroxyurea by more than 20% in the past 90 days (if applicable)
  • Have not changed their dose of crizanlizumab or L-glutamine for the past year, unless it was necessary because of a change in weight (if applicable)
People who do not use hydroxyurea, crizanlizumab, or L-glutamine can still be eligible for the study, the conditions described above are only for people who do use these therapies. For more information about this study, please contact the study team at lindsey.ford@duke.edu.

Age Range

18-65

Sex/Genders

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

What is Involved?

Description

This study is made up of 2 parts: the double-blind drug period and the open-label extension. If you choose to join this study, during the double-blind drug period you will get a random assignment (like a coin flip) to 1 of 2 groups:
  • One group will get the study drug
  • The other group will get a placebo ("sugar pill" that has no drug in it)
Participants in both groups will:
  • Have blood draws
  • Have physical exams
  • Have heart scans (ECG)
  • Fill out surveys
The double-blind drug period will last for 1 year. During this time, you will take the study drug or placebo one time every day. The study drug or placebo are two tablets that you will take by mouth. During the double-blind drug period, neither you nor anyone on the study staff will know if you are taking the study drug or placebo. However, if there is a medical need to know this information, we can find this out and your study participation will end. When you finish the double-blind drug period, you will have the option of joining the open-label extension. Anyone who joins this period of the study will be guaranteed to get the study drug regardless of whether or not you got the study drug in the double-blind period. The open-label extension period will last for 112 weeks. There is a brief video about this study that you can watch online: https://vimeo.com/604234244/5f73735a28

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

No

Study Details

Full Title

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease

Principal Investigator

Marilyn
Telen

Protocol Number

PRO00106730

NCT ID

NCT04624659

Phase

II/III

Enrollment Status

Open to Enrollment
ClinicalTrials.gov