Sickle cell disease: Useful research terms and resources

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You may be asked to join a clinical research trial or other types of research to help research teams study new treatments and better understand the impact of sickle cell disease on families. Learn about types of research and where to find more information about sickle cell disease research.

Key points

  • If you are asked to take part in sickle cell disease research, you may hear or see these research terms: clinical trials, standard therapy, trial phases.
  • Clinical trials are research studies that help research teams see how sickle cell disease affects the body, how to treat sickle cell disease and how to make patients more comfortable. They are the best way to figure out if a new treatment, medicine or a mix is better than what is already available.
  • Standard therapy is the current best treatment, which is the treatment people with sickle cell disease normally get when they are not part of a clinical trial.
  • New treatments go through phases of testing, which are reviewed many times by experts to protect the rights and safety of the patient. Teens most commonly join Phase III trials (the final phase).

When you are asked to get involved in research, you will come across some specific research terms. These include:

Clinical trials

Clinical trials are research studies that aim to improve the quality of life of patients with sickle cell disease. They can be designed to study how sickle cell disease affects the body, how to treat sickle cell disease and how to make patients more comfortable.

When you enroll in (join) a clinical trial, a team will study you over time to see:

  • how well a treatment works
  • if a treatment is safe
  • what kind of side effects a treatment might cause

Clinical trials are the best way to figure out if a new treatment, medicine or mix of both is better than what is already available.

Standard therapy

In clinical trials, new treatments are compared with standard therapy. Standard therapy means the therapy that is the current best treatment. It is the treatment that patients normally get when they are not part of a clinical trial.

Trial phases

New treatments go through phases of testing. Before each phase of a trial is started in a hospital or clinic, it is reviewed many times by many different experts to make sure that the safety and rights of patients are protected.

Every treatment is first tested in a laboratory. If the treatment seems to work, it will move to a phase 1 trial, then phase 2 and, finally, phase 3.

Phase I (1): This type of trial aims to figure out the highest dose of a treatment that can be given with acceptable side effects. Groups of study participants start off with a low dose. The dose is slowly increased until the side effects they experience are unacceptable.

Phase II (2): Based on the results of phase 1, the treatment is now given to patients. The study looks at how the body and sickle cell disease respond to the treatment and whether the proposed dose is safe to use.

Phase III (3): The treatment is now compared with standard therapy to see if it is better in some way (for example, if it reduces how often a pain crisis happens, has reduced side effects or makes hospital stays shorter). Teenagers are most commonly enrolled in Phase III trials.

Other types of research

You and/or your parent(s)/caregiver(s) may be asked to enroll in research that is not part of a clinical trial or is not related to testing a medical treatment. This type of research is also important. It can help health-care professionals understand the impact of sickle cell disease on the lives of patients and families.

In other types of research, you or your parent(s)/caregiver(s) may be asked to answer questions in an interview, fill in a survey, try out a new technology or be part of a new program to help patients and families cope with sickle cell disease.

Taking part in research like this helps people with sickle cell disease and their families get better support from the health-care system.

Where to find more information about research

Children and clinical studies

To learn more about taking part in a research study, check out this link to watch some video clips about the experiences of other children and teens. This website was created by the National Institutes of Health, a government agency in the United States that provides leadership and support for research studies.

ClinicalTrials.gov – Sickle cell anemia

The ClinicalTrials.gov website is a database of publicly and privately supported clinical research studies from around the world. Managed by the National Institutes of Health in the United States, it provides up-to-date information to patients, their families and caregivers, health-care professionals and the public. The link above takes you straight to the listing of research studies on sickle cell disease and sickle cell anemia.

Last updated: November 6th 2023