Whether you are a patient, a family member, or a caregiver, if you are hunting for clinical trials on sickle cell disease, you no longer have to scour the web or hound your healthcare provider. You can now search for sickle cell disease clinical trials suitable for you by location, phase, study type, or relevant keywords through an easy-to-navigate, centralized website launched by the Sickle Cell Disease Association of America (SCDAA) on Oct 13, 2021.

Funding to develop the platform came from a partnership between Forma Therapeutics and the SCDAA.

“Until now, people with sickle cell disease had to search far and wide to find clinical trials. Patients would ask their physicians, call medical systems, or search through government sites such as the NIH clinical trial finder—which sickle cell disease patients have found to be cumbersome to navigate and not easy to find trials in close proximity to where they live or work,” said Beverley Francis-Gibson, president and CEO of the SCDAA, and who herself has lost a family member to the disease.

“The clinical trial finder provides a one-stop-shop for patients and their families to discover, learn about, and connect with trials that are critical to discovering therapies and treatments for sickle cell disease.”

Typically, patients search for trials close to them so that they can access the treatment, testing, monitoring, and follow-up during their participation in a trial. “Our emphasis now is to focus on trials in the United States and eventually expand internationally,” said Francis-Gibson.

Currently, the clinical trial finder connects to 169 clinical trials on sickle cell disease. While the study centers are located around the world, the trials themselves are based in the United States.

“This tool was developed in response to the frustrations we have been hearing from our patients and the pharmaceutical companies running the trials. Our goal was to remove any barriers to participation by creating an easy-to-use clinical trial finder site that serves as the hub for sickle cell disease patients to seamlessly connect to trials by allowing them to search by proximity to them, type of trial, and study topic, and then communicate directly through the site with the trial coordinators,” said Francis-Gibson.

In healthy individuals, flexible, disc-shaped, red blood cells wind through fine vessels shuttling oxygen-loaded hemoglobin to every living cell in the body. However, in sickle cell disease, mutations in the beta-globin gene lead to the synthesis of faulty hemoglobin proteins that change flexible red blood cells into inflexible, sickle-shaped cells that block blood flow causing pain and organ damage.

Currently, there is no universal cure for sickle cell disease.

Before a new treatment can be made available widely in the consumer market, a manufacturer needs to acquire approval through several steps and stages that test the safety and efficacy of the new treatment. These interventional or observational, health-related research studies in humans that follow a defined protocol are called clinical trials. Whereas interventional studies involve the administration of experimental treatments on trial participants, observational studies involve measuring outcomes in participants who do not necessarily receive a treatment.

Through clinical trials that are conducted over several phases, pharmaceutical companies collect data about the safety and efficacy of treatments and can provide participants new treatments before they become widely available. Clinical trial teams include doctors, nurses, social workers, and other health care professionals who monitor the health of the participants at regular intervals throughout the trial and provide specific instructions for participating in the trial.

Every year, thousands of patients participate in clinical trials across the globe, to either gain access to new treatments at the cutting edge of science that are not yet widely available or to contribute to medical science and help others. Several clinical studies provide participants compensation for time and travel expenses.

In addition to the clinical trial finder, the SCDAA also provides a Sickle Cell Disease Educational Information Repository (SCD Repository) with an up-to-date directory of information and resources on sickle cell disease. The organization advocates for patients and families suffering from sickle cell disease and helps community-based organizations raise public awareness and advance the search for a universal cure.

Francis-Gibson said, “The educational content offered on the site provides a better understanding of clinical trials for those considering participation. By streamlining and simplifying this process and educating our patients, we believe trial participation will significantly increase. To further bolster this effort, we are launching a large marketing campaign to generate awareness of this tool with sickle cell disease warriors and their caregivers as well as the clinical community and our member organizations throughout the United States.”

Through efforts of the SCDAA, member organizations, and pharma companies, patient voices from diverse ethnic communities are being represented in clinical trials that are working toward improved services, treatment, legislation, and a cure for this silent killer.

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