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



Status:Recruiting
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - 100
Updated:4/6/2019
Start Date:April 10, 2019
End Date:August 31, 2019
Contact:Hae L Cho
Email:hae.cho@nih.gov
Phone:(301) 496-2429

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Background:

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.

Objective:

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

Eligibility:

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.

Design:

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.

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.

- INCLUSION CRITERIA:

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.

EXCLUSION CRITERIA:

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
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
301-496-2563
Phone: 800-411-1222
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
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Bethesda, MD
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