Active not recruitingPHASE3INTERVENTIONAL

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

This important study, called HIBISCUS, is looking into a new tablet medicine, etavopivat, for individuals aged 12 to 65 living with sickle cell disease. Researchers want to find out if etavopivat can help improve the amount of a protein called haemoglobin in the blood and reduce the number of painful events known as vaso-occlusive crises, where blood vessels get blocked. We will be comparing different doses of the medicine against a dummy pill (placebo) to see how effective and safe it is. If you have sickle cell disease and experience these crises, this study could be an opportunity to explore a potential new treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Forma Therapeutics, Inc.

Enrolment target

450

Start

29 Jan 2021

Estimated completion

19 Mar 2027

Sickle Cell Disease

What is this study about?

Sickle cell disease is a lifelong condition that can cause severe pain and other problems. This clinical trial is investigating a new tablet medication called etavopivat. The main goal is to see if this medicine can make a real difference for people with sickle cell disease, specifically by improving their red blood cells and hopefully leading to fewer painful episodes where blood vessels become blocked. These painful events are often called 'crises'.

The HIBISCUS study will compare etavopivat against a 'placebo', which is a dummy pill that looks just like the study medicine but contains no active drug. This helps the researchers understand if any improvements are genuinely due to the medicine or just a natural change. Participants will not know if they are receiving the active medicine or the placebo, and neither will their doctor, until the study is over. This is called a 'double-blind' study and helps ensure the results are accurate and unbiased.

Initially, some participants will receive one of two doses of etavopivat, while others will receive the placebo. Later, based on early results, the study will focus on the most promising dose of etavopivat compared to the placebo. After taking the study medicine for about a year, participants might have the option to continue taking etavopivat for an even longer period through an 'open-label' extension, where everyone involved would know they are receiving the active medicine.

Key takeaways

This study is for people aged 12-65 with sickle cell disease.
It tests a new tablet called etavopivat to improve red blood cells and reduce pain.
Participants will receive either the medicine or a placebo (dummy pill).
The study lasts about 1 year, with an optional 2-year extension.
Regular health checks and blood tests will be part of the process.
Your decision to join or leave the study won't affect your regular medical care.

Who may be eligible?

To join this study, you need to be aged between 12 and 65 years old and have a confirmed diagnosis of sickle cell disease. You should have experienced at least two painful crises (vaso-occlusive crises) in the last year. Your haemoglobin levels, which measure a protein in your red blood cells, need to be within a specific range when you are checked for the study.

There are also some requirements if you are already taking other medicines for sickle cell disease. For example, if you take hydroxyurea, you need to have been on a stable dose for at least 3 months. If you are on crizanlizumab or L-glutamine, you need to have been on a stable dose for at least 12 months and be taking it regularly.

Certain health conditions or situations would mean you cannot take part. For instance, if you have had more than 15 painful crises in the last year, are pregnant or breastfeeding, have certain severe liver or kidney problems, or have uncontrolled heart or lung conditions. Also, if you have HIV or active hepatitis B or C, or have received regular blood transfusions, you would not be able to participate.

Quick self-check

Are you between 12 and 65 years old?
Do you have a confirmed diagnosis of sickle cell disease?
Have you had at least 2 painful sickle cell crises in the past year?
Do you have certain stable haemoglobin levels (checked by the study doctor)?
Are you able to use effective contraception if you are a woman of childbearing potential, or a barrier method if you are a man?
Are you not currently receiving regular blood transfusions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be given tablets to take. You might receive one of two different doses of the new medicine, etavopivat, or a dummy pill (placebo). Neither you nor your doctor will know which you are receiving during the main part of the study. You will take these tablets for about 52 weeks (roughly one year).

Throughout this year, you will have regular visits to the clinic so the study team can monitor your health, take blood tests to see how the medicine is affecting you, and check for any side effects. After this first year, if you wish and if the study team agrees, you may have the option to continue taking etavopivat for an additional 112 weeks (about two years) in what's known as an 'open-label' period, where everyone will know you are receiving the active medicine. The exact number of visits and tests will be explained in detail by the study team.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as receiving a new medicine before it's widely available and contributing to medical research that could help others with sickle cell disease. However, there are also potential risks; the new medicine might cause side effects, or it might not work for you. As with any medicine, some side effects could be serious. You will be closely monitored throughout the study for your safety. It's important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (142)

Univ of Alabama Birmingham
Birmingham, United States
Phoenix Children's Hsptl
Phoenix, United States
[Legal] Woodland International Research Group, LLC
Little Rock, United States
[Legal] Collaborative Neuroscience Network, LLC
Long Beach, United States
Pacific Research Partners
Oakland, United States
UCSF Oakland Benioff ChildHosp
Oakland, United States
University Of California Irvine
Orange, United States
UC Davis Medical Center
Sacramento, United States
University of Connecticut
Farmington, United States
Yale University
New Haven, United States
Children's National Health Hospital
Washington D.C., United States
Howard University
Washington D.C., United States

+130 more sites — see the official record for the full list.

Common questions

What is sickle cell disease?

Sickle cell disease is a genetic condition where your red blood cells are abnormally shaped, like a crescent moon. This can cause them to block blood vessels, leading to pain and other health issues.

What is a 'placebo'?

A placebo is a 'dummy' pill that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare if the new medicine is truly effective.

What are 'vaso-occlusive crises'?

These are painful episodes that happen when the sickle-shaped red blood cells block small blood vessels, stopping blood flow to different parts of the body.

Will I know if I'm getting the real medicine or the placebo?

No, during the main part of the study, neither you nor your doctor will know if you are receiving the active medicine or the placebo. This is called 'double-blind'.

How long will I be in the study?

The main part of taking the study medicine will last about one year (52 weeks). After that, you may have the option to continue for another two years in an open-label part of the study.