DREAM is a clinical trial for 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 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 have been diagnosed with Sickle Cell Disease with genotype HbSS or HbSB.
This study is being conducted in 2 parts (A and B). Part B is currently enrolling:
- Part B will include adult participants aged 18 years and older.
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 this clinical trial. If you choose to enroll yourself, the study staff will check that all the requirements are met during a screening visit.
Representation in study participants is important. This helps scientists learn how potential medicines work in different people. Factors like genetics, race, ethnicity, age, and sex can all impact how people respond to the same treatment. That’s why it is important that the DREAM study includes people of all backgrounds with Sickle Cell Disease.
Condition
Sickle Cell Disease
Age
18 years and older
Sex
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.
What to expect
Part B of this study is 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 whether a participant is receiving the study medicine or placebo. During this clinical trial’s treatment period participants will attend about 12 clinic visits and have phone calls between visits. Visits will include assessments such as blood tests and questionnaires.
Upon completion of treatment in Part B, participants may have the option to join an extension study, in which all participants will receive the study medicine.
Length of study treatment
48 weeks
Number of study visit
14 visits and 14 phone calls
Long-term follow up
12-week follow-up or an optional extension study to continue the study medicine
About the study treatments
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. The study medicine (or placebo) will generally be taken once per day during the treatment period.
Frequently asked questions
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 vaso-occlusive, or pain crises.
Participants will be allowed to take standard medications for disease management or pain crises, if needed during the study, with certain exceptions 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.
Participants may have the option to join an ‘open-label extension’ (OLE) study after taking part in the DREAM clinical trial. Before taking part in the extension study, participants will need to sign the OLE section of the informed consent form, if they have not done so already. All participants in the extension study will receive the study medicine as one daily tablet to be swallowed whole with water – there will be no placebo group.
Taking part in the DREAM study may help people with SCD in the future by increasing our understanding of the study medicine.
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.
On average, study visits in Part B will last about 1-2 hours. However, there are also some longer study visits, including one visit that will last about 4 hours.
Yes, the decision to take part is entirely the participant’s choice. If you decide to 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 regular medical care or any benefits to which you are entitled.
Participation will not affect regular medical care in the future. If you stop taking part, you 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.
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 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.
You will be reimbursed for any reasonable expenses that you may have as a result of taking part in this study. This may include parking, meals, or other travel-related expenses.
All information collected from this study will be kept private and will not personally identify you. Security measures will be used to protect your data against accidental or unlawful loss, alteration, or unauthorized disclosure or access.
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.
