DREAM is a clinical trial for children and adults with Sickle Cell Disease (SCD). This clinical trial will explore if a study medicine can reduce symptoms and improve the overall health of people with SCD.

Thank you for learning more.

SCD impacts about 100,000 Americans and is the most common inherited blood disorder in the US. People diagnosed with SCD can experience worse health outcomes and a shorter life expectancy than the average American.

When taking part in a clinical trial, you (or your child) will receive attentive care from a team of healthcare providers and researchers.

Participation in the DREAM study could lead to improvements in the treatment of SCD and how it’s managed for current and future generations.

Who may participate

Thank you for taking the time to learn more. The DREAM study may be an option if you or your child have been diagnosed with Sickle Cell Disease.

This study is being conducted in 3 parts (A, B & C). Parts B and C are currently enrolling:

  • Part B will include adolescent and adult participants aged 12 years and older.
  • Part C will include children aged 6 months to 17 years. Older children will be enrolled first, then younger children and infants will be enrolled.

A person with SCD will not be able to participate in this study if they:

  • Have had more than 10 vaso-occlusive crises (VOCs, sudden painful crises) in the last year
  • Are receiving regularly scheduled blood transfusion therapy
  • Are pregnant or breastfeeding

There are other requirements to take part in DREAM. If you choose to enroll yourself or your child, the study staff will check that all the requirements are met during a screening visit.

Many factors, including genetics, race, ethnicity, and gender, can impact how people respond to a medicine. It is important that DREAM includes people of all backgrounds with SCD.


Sickle Cell Disease


Part B: aged 12 years and older & Part C: aged 6 months to 17 years


Male or Female

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.

Get started – See if you (or your child) may be eligible

Answer a 2-minute questionnaire.

A first step as you consider this study is to answer a 2-minute online questionnaire about your (or your child’s) health and medical history. If your answers show the study might be a good fit, you may choose to have your contact information shared with a Pfizer Clinical Trial Contact Center Navigator.

Speak with a study representative.

If you’re interested, a Pfizer Clinical Trial Contact Center Navigator will call you and do a more detailed review of the study and your (or your child’s) medical history to see if you (or your child) may be able to participate and be referred to a study clinic that you select.

Confirm your eligibility.

Your answers to these questions will only be linked to you (or your child) if your responses indicate that you (or your child) may be eligible to participate in this study and you choose to share your contact information with the study team. Pfizer study team members and our partners will have access to reports containing aggregated data that will not be directly linked back to individuals. Only the study staff can determine if you (or your child) meet the study’s eligibility criteria and are able to enroll in the study.

What to expect

Part B and Part C of this study are currently enrolling participants.

Part B will investigate the effects of the study medicine on hemoglobin levels and other symptoms of SCD. Participants will take the study medicine or placebo (which looks like the study medicine but does not contain any active medicine) for 48 weeks. Neither the participants nor the study team will know which treatment a participant is receiving. During the treatment period participants will attend about 11 clinic visits and have phone calls between visits. Visits will include assessments such as blood tests and questionnaires.

Part C will research how the body processes the study medicine in children. Participants will start by taking a single dose of the study medicine. An independent committee will then monitor their health and blood tests for 7 weeks and decide what dose is right for them. They will then take the study medicine for 2 weeks and be monitored for a further 12 weeks.

Length of study treatment

Part B: 48 weeks & Part C: single dose + 2 weeks

Number of study visit

Part B: 13 visits and 15 phone calls & Part C: 15 visits

Long-term follow up

12-week follow-up or an optional extension study to continue the study medicine

About the study treatments

Portrait of an African American family – Pfizer Clinical Trials
Portrait of an African American family – Pfizer Clinical Trials

About the study treatments

People with Sickle Cell Disease have abnormal hemoglobin (the part of red blood cells that carries oxygen). This changes the shape of the cells from a disc to a crescent or “sickle.” Sickle cells are prone to blocking blood vessels and the abnormal hemoglobin can also damage red blood cells.

The study medicine is thought to work by helping the hemoglobin in red blood cells hold on to more oxygen, which keeps them in their normal shape and better able to deliver oxygen to the body.

The study medicine (or placebo) is taken by mouth with a glass of water, usually as a tablet. If the participant is not able to swallow a tablet, another formulation will be available. The study medicine (or placebo) will generally be taken once per day during the treatment period.

Potential benefits and risks of taking part in the DREAM study

People who take part in clinical studies are key to advancing medical research. By taking part in the DREAM study, you/your child will be aiding medical research which may help others with SCD in the future.

Over the course of the study, a participant’s health may get better, get worse, or stay the same. People who are considering taking part in the study will be given a complete list of risks and possible discomforts before agreeing to participate. As with any medication, there may be a reaction to the study medicine.

Potential benefits and risks of taking part in the DREAM study

Middle-Eastern family eating at the dinner table – Pfizer Clinical Trials
Middle-Eastern family eating at the dinner table – Pfizer Clinical Trials

Frequently asked questions

What is Sickle Cell Disease (SCD)?

Sickle Cell Disease is an inherited blood disorder. This means that SCD is passed through genes from parents to children. People with SCD have abnormal hemoglobin (the part of red blood cells that carries oxygen), called sickle hemoglobin in their red blood cells. This causes low blood counts (anemia) which can cause lower energy levels and can also damage blood vessels - leading to problems like strokes and leg ulcers. The lack of oxygen in body tissues due to poor blood flow can cause attacks of sudden, severe pain, called pain crises.

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Can participants in DREAM keep taking other medicines for SCD?

Study participants will be allowed to take other medications as needed, with certain exceptions that the study team can explain to you.

Participants taking hydroxyurea (HU) and L-glutamine can continue these treatments while in the DREAM study, but they must be on a stable dose for at least 90 days prior to joining the study.

Medications for SCD that are not allowed during the study are voxelotor, crizanlizumab and erythropoietin (EPO).

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How long do study visits last?

On average, study visits in Parts B & C will last about 1-2 hours. However, in Parts B & C there are also some longer study visits, including one visit that will last about 4 hours and one visit that will last about 8 hours.

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Is taking part in this clinical trial voluntary?

Yes, the decision to take part is entirely the participant’s (or their parent/guardian’s) choice. If you decide to participate (or have your child participate), you will be free to stop taking part at any time and for any reason. A decision to stop being in the study will not affect your (or your child’s) regular medical care or any benefits to which you are entitled.

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How will participation affect future medical care?

Participation will not affect regular medical care in the future. If you/your child stops taking part, you/they may be asked to return to the clinic if needed for an ‘end-of-study assessment’. Participants with ongoing medical issues may be followed until the issue is resolved or medically stable.

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Do participants have to pay to be in the study? Is health insurance needed to participate?

The study medicine and any study-related procedures are covered at no cost. Participants do not need health insurance to take part.

However, if you are considering joining or having your child join Part B of this study, you should talk with your insurance company about its payment policy for standard medical care given during a research study, including any treatment needed for possible side effects from the study. If your insurance company does not pay, you may be billed for those charges.

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Is there travel support available for participants in this study?

You will be reimbursed for any reasonable expenses that you may have as a result of taking part or having your child take part in this study. This may include parking, meals, or other travel-related expenses.

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Will my personal information be kept private?

All information collected from this study will be kept private and will not personally identify you (or your child). Security measures will be used to protect your data against accidental or unlawful loss, alteration, or unauthorized disclosure or access.

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Why do we need clinical trials?

A clinical trial (or study) is a type of medical research in which people volunteer to take part. Clinical trials are the best way to investigate how potential medicines and other medical treatments affect patients. All medicines that become available today for use in people are first tested in studies involving hundreds to thousands of participants.

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