An observational study in Sickle Cell Disease, sponsored by National Institutes of Health Clinical Center (CC). Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-09-01.
Sponsored by National Institutes of Health Clinical Center (CC) · Observational
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:
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.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 27 is below the median of 100 across 287 observational studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →National Institutes of Health Clinical Center (CC) is the lead sponsor of 367 studies on the registry; 27 are open to participants now.
Of its 22 completed or terminated interventional studies of FDA-regulated products, 18 (82%) have results posted.
Counted across the registry records on this site, refreshed daily.
Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH
Our inclusion criteria for study subjects are:
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:
EXCLUSION CRITERIA:
Study subjects will be excluded if they:
Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy
Expectations, understanding and motivations for enrollment
Understanding, expectations, motivations, and decision making process
Time frame: Before or after enrollment in PBSCT or GT trial, but after decision made
Explore the role of family and religion
Description of the role of family, culture, and religion in enrollment decision making
Time frame: After decision made about enrollment
This study is completed, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.
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National Institutes of Health Clinical Center (CC)