To determine the efficacy and safety of a new medication or treatment, clinical research is necessary. Clinical trials vary based on the research team’s goals, but their purpose remains the same: to improve overall health outcomes for the public. However, the criteria for who can join a clinical trial may vary.
Sickle cell disease (SCD) is a genetic blood disorder that affects over 100,000 people in the United States and is particularly prominent in Black patients. Over 90 percent of SCD cases are found in non-Hispanic Black individuals. Despite this, the Black community continues to be underrepresented in clinical trials.
Not every patient with SCD will qualify for every SCD clinical trial. Each trial has its own eligibility requirements to help researchers determine whether a study is safe and whether participants fit the research question.
Here’s what you need to know about joining a sickle cell clinical trial.
Eligibility criteria vary by SCD clinical trial. If a participant doesn’t meet the study’s specific criteria, they may be denied or advised to seek another study.
Common criteria for an SCD clinical trial include:
Sheinei Alan, MD, PhD, is the Director of the Adult Sickle Cell Program and Director of Clinical Research at the Inova Schar Cancer Institute, located in Fairfax, Virginia. Dr. Alan notes that eligibility for a sickle cell trial will be based on “the aim and population of interest.”
“In sickle cell disease, this will be based on genotype (often HbSS and HbSβ⁰ only), age, hemoglobin range, kidney and liver function, chronic transfusion status, stroke history, stable hydroxyurea dose, and vaso-occlusive crisis frequency,” Dr. Alan told BlackDoctor. “This last criterion is a major barrier in recruitment because it often selects between 2-10 events per year, which many patients do not meet.”
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Clinical trial eligibility requirements protect participants’ health and safety and help answer the research question as accurately as possible. Some trials may require a specific number of pain episodes in the past year, while others may only require lab values, such as a white blood cell count.
According to Dr. Alan, eligibility requirements allow the removal of “confounding variables that could contribute to the results, improve statistical power, ensure safety, and answer the question it set out to answer.”
Other trials might be analyzing the effect of a medication on a specific population, like children, requiring different age restrictions. Some applicants may have other health conditions that, when in conjunction with a new treatment, could put their health in jeopardy. The researchers’ main goal is to protect patient health.
Every clinical trial has its own inclusion and exclusion criteria, which determine who can and cannot participate. According to a 2018 report from the Food and Drug Administration (FDA), these criteria “are often tailored to allow assessments of the effectiveness of a treatment in a well-defined population.” Researchers often try to balance specific criteria with a broad participant population to help as many people as possible.
Inclusion criteria, as defined by the FDA, “specify the characteristics required for study entry.” This can include specific demographics, stage of disease, age range, a certain mutation of the disease, gender, current medications, etc.
The FDA defines exclusion criteria as “characteristics that disqualify patients from participation.” This can include too-late or too-early stage of disease, falling outside the required age range, organ dysfunction, pregnancy, and even factors like location and finances.
If you’ve applied but been denied participation for a clinical trial, you can still join another one. All clinical trials will have specific eligibility requirements, and being disqualified for one trial doesn’t mean there aren’t other opportunities to participate.
“Many patients fall outside of eligibility criteria for sickle cell clinical trials,
Dr. Alan said. “Ineligibility describes the protocol, not the patient, and it is worth saying that plainly because patients hear it as a verdict on their disease.”
If you or a loved one are hoping to find an SCD clinical trial, here are a few resources to consider:
Once you’ve sourced a potential SCD clinical trial, here are some common steps to take:
Meeting eligibility criteria will not automatically guarantee your acceptance into the trial. Ultimately, researchers will make the final decision on participants.
If you qualify for the study, you will move on to signing informed consent.
Since Black Americans make up an overwhelming percentage of sickle cell disease cases in the U.S., this community needs to be represented in clinical trials for the condition. When researching a treatment or medication for a disease, researchers need participants from the communities that are most impacted by the disease being studied.
“Representation is incredibly important because sickle cell remains to be well studied and treated,” Dr. Alan says. “We need more therapies and tools in our toolbox. Sickle cell disease has historically received inadequate funding.”
While participating in a clinical trial may come with risks, it can also come with benefits, such as:
RELATED: 5 Black Clinical Trials FAQs, Answered
If you or a loved one have been diagnosed with SCD and are interested in participating in clinical research, there are many clinical trials for which you may be eligible. Not only could you gain access to innovative treatment and care, but you would also represent your community in medical research.
If you’re unsure of where to start when it comes to searching for an appropriate clinical trial to take part in, you can visit BlackDoctor’s Clinical Trials Resource Center to view open trials.
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