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Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03709303
Recruitment Status : Recruiting
First Posted : October 17, 2018
Last Update Posted : September 19, 2019
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC)

Brief Summary:


Sickle cell disease is an inherited blood disorder. People with this disease have a problem with their hemoglobin. That is a protein in red blood cells that carries oxygen in the body. Some people with this disease are enrolled in research at NIH. Researchers want to learn more about the thoughts and opinions of those people. This may improve the way researchers explain clinical studies, risks, and benefits to people with the disease.


To learn about the motivations, decisions, and experiences in clinical research of people with sickle cell disease.


Adults ages 18 and older who have sickle cell disease. They must be in an NIH study on this condition. They must have been invited to join either a gene therapy or peripheral blood stem cell transplantation study.


Participants will have 1 interview. It will be done in a quiet room in the NIH Clinical Center or by video call. It will take about 60 minutes.

The interview will be audio-recorded if the participant agrees.

Participants will be asked about:

  • Their experiences with and thoughts on sickle cell disease
  • Their decision to participate in clinical research
  • Factors that may have affected their decision to participate. These may include family, disease history, or faith.

Participants may complete a few brief questionnaires.

Condition or disease
Sickle Cell Disease

Detailed Description:

Clinical trials testing potentially curative interventions for sickle cell disease - such as gene therapy (GT) or peripheral blood stem cell transplantation (PBSCT) - have created a novel opportunity for patients with sickle cell disease, for whom standard therapies can only manage but not cure their conditions. But some of these experimental interventions may pose risk of significant adverse events. As the development of these interventions create a new decision-making situation for persons with longstanding diseases, these trials raise questions about the expectations and decision- making process of patients considering and/or participating in them. Given the longstanding debate in the bioethics literature about whether patients expressions of desire for benefit are (1) evidence of false hopes or poor understanding of risks and benefits, or, instead (2) expressions of natural optimism compatible with valid informed consent, these patients present an opportunity to learn about how they make their participation decisions which in turn could inform this longstanding debate.

This study aims to explore the expectations, understanding, motivations, and decision-making of patients with sickle cell disease who have either chosen or declined to enroll in research studies testing experimental interventions. The aim is to better understand how these patients understand the research study, assess risks and benefits at the time of enrollment, make decisions, and react to their health outcomes. As faith and religion are known to play an important part in the lives of persons with sickle cell disease, we will also explore the role of religion and faith in sickle cell patients decision-making and retrospective perspective on their decision.

Primary Hypothesis:

This is a descriptive, explorative study. It may generate hypotheses for future studies.

Purpose of the Study Protocol:

To describe how patients with sickle cell disease understand and make decisions about participating in gene therapy (GT) or peripheral blood stem cell transplantation (PBSCT) clinical trials to shed light about the ethically salient issues regarding enrollment and participation in high- risk, high-reward clinical trials. This information may inform practices around the informed consent process and help researchers better understand the role of family and religion/faith in clinical research decision making.

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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Case-Control
Time Perspective: Cross-Sectional
Official Title: Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research
Estimated Study Start Date : September 24, 2019
Estimated Primary Completion Date : August 31, 2020
Estimated Study Completion Date : August 31, 2020

Resource links provided by the National Library of Medicine

SCD patients
Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy

Primary Outcome Measures :
  1. Expectations, understanding and motivations for enrollment [ Time Frame: Before or after enrollment in PBSCT or GT trial, but after decision made ]
    Understanding, expectations, motivations, and decision making process

Secondary Outcome Measures :
  1. Explore the role of family and religion [ Time Frame: After decision made about enrollment ]
    Description of the role of family, culture, and religion in enrollment decision making

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH

Our inclusion criteria for study subjects are:

  • Adult (18+ years and older)
  • Sickle cell disease patients who are enrolled in at least one of the following sickle cell disease protocols at the National Institutes of Health, National Heart, Lung, and Blood Disease:

    • Screening study: 08-H-0156
    • Natural history protocol: 04-H-0161
    • PBSCT: 09-H-0225, 17-H-0069, 14-H-0077, 03-H-0170
    • GT: 14-H-0155
  • Have made a decision regarding participation in one of the GT or PBSCT protocols (e.g. someone could still be enrolled in Screening study, have made a decision, but not yet enrolled in or have declined enrollment in the GT or PBSCT studies)
  • Study subjects will be recruited in two groups: 1) pre-transplant or pre-initiation of gene therapy, including both those who decide to enroll and those who declined to enroll; and 2) post-transplantation or post-GT, including those who had an unsuccessful and those who had a successful transplantation or response to GT.


Study subjects will be excluded if they:

  • Lack cognitive capacity
  • Are not English speaking
  • Have not made a decision about participation in one of the GT or PBSCT protocols for which they are eligible

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03709303

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Contact: Hae L Cho (301) 496-2429

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United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010   
Sponsors and Collaborators
National Institutes of Health Clinical Center (CC)
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Principal Investigator: Christine Grady, Ph.D. National Institutes of Health Clinical Center (CC)

Additional Information:
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Responsible Party: National Institutes of Health Clinical Center (CC) Identifier: NCT03709303     History of Changes
Other Study ID Numbers: 190004
First Posted: October 17, 2018    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: October 15, 2018
Keywords provided by National Institutes of Health Clinical Center (CC):
Informed Consent
Stem Cell Transplantation
Sickle Cell Disease
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn