Because living with sickle cell disease should be made easier.

This is a Phase 1 study evaluating the safety and effectiveness of an investigational medicine to prevent painful episodes caused by sickle cell disease (SCD) called vaso-occlusive crises (VOCs). Adults ages 18 to 65 years old with sickle cell disease who have been seen by a healthcare provider for 2 to 10 VOCs in the past year may be eligible. Learn more to see if you can participate.

Card Summary Component

Who may participate

Joining a clinical trial is an important and personal decision. Your doctor may have mentioned this study to you, or perhaps you found your way here by yourself. Either way, thank you for your interest. 

This study may be an option if you

  • are 18 to 65 years old  
  • have been diagnosed with sickle cell disease 
  • have had 2 to 10 VOCs in the past 12 months requiring contact with a healthcare provider (e.g. emergency department, hospitalization, clinic visit etc.)

There are other requirements for taking part in this study. The study doctor will explain these to you.


Sickle Cell Disease

The disease, disorder, syndrome, illness, or injury that is being studied. On, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.


18 to 65 years

The age a person must be to participate in a clinical trial.


Male or Female

The sex of people who may participate in a clinical trial. Sex is a person’s classification based on assignment at birth. Eligibility based on sex is distinct from eligibility based on gender.

Each clinical study has its own guidelines for who can participate, called eligibility criteria. However, only the research study staff can determine if you qualify to enroll in the study.

Card Summary Component

What to expect

If you are interested in participating, the study team will schedule a screening visit to determine if you can join the study. 

If you are eligible for the study and decide to participate, you will receive the study medication once a month for three months. Doses will be given as an injection under the skin.   

You will be enrolled in the study for about 5 months. During this time, you will need to attend 9 in-clinic appointments and 2 virtual interviews so the study team can monitor how you are responding to the study medication. These visits may include physical exams, questionnaires, blood samples, and ECGs. 

You will also need to complete daily entries in an electronic diary to track your SCD symptoms and how they are affecting your usual activities. These entries will take about 3-5 minutes every day.

Length of study treatment

3 Months

Length of study treatment is the length of time the study participants will receive the study treatment.

Number of study visits

9 in-person visits; 2 virtual interviews

Each clinical trial’s design specifies the number of study visits and the total length of the trial.

Long-term follow up

2 months after last dose

Some studies require the study team to stay in contact with the participant for a period of time after the participant completes the main part of the study. This long-term follow up is to collect additional information on the study drug over time.

About sickle cell disease

Sickle cell disease (SCD) is a genetic (inherited) disease that causes red blood cells to be shaped like a crescent or half-moon instead of round. These abnormally shaped cells tend to get stuck in small blood vessels, which blocks blood flow and causes severe pain. People with SCD may also have a chronic shortage of red blood cells (anemia) that causes tiredness, weakness, and difficulty breathing.

About vaso-occlusive crisis

Vaso-occlusive crisis (VOC), or sickle cell crisis, is a painful complication of SCD. VOCs occur when abnormally-shaped red blood cells get stuck and clog small blood vessels, blocking blood flow to certain parts of the body. These blockages cause severe pain and may lead to organ damage from lack of oxygen. 
Although there are currently some treatments available, most people with SCD continue to experience VOCs. New treatment options are needed to improve the lives of people living with SCD by reducing the duration and severity of VOCs.

Frequently asked questions

How long will I be in the study?

Participation in this study will take approximately 5 months (including the time it takes to complete screening).

Is this page helpful?

What if this study is not right for me?

You can choose to leave the study at any time, for any reason. Choosing to leave the study will not affect your SCD treatment or future medical care.

Is this page helpful?

Am I guaranteed to receive the study medicine?

Yes, all participants will receive 3 doses of the study medicine. This study does not include a placebo (no active ingredient).

Is this page helpful?

What are the possible side effects?

All medicines have the potential to cause unwanted side effects. The study doctor will discuss the possible risks and benefits of participating in this study with you before you choose to enroll. You can choose to leave the study at any time.

Is this page helpful?

Does it cost anything to participate?

No. Participants will receive all study-related tests and procedures for free. Additionally, participants will receive reimbursement for attending study visits and completing daily entries in the electronic diary. Transportation and nearby lodging can also be arranged at no cost.

Is this page helpful?