Sickle Cell DiseaseNCT03709303
Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research
An observational study of Sickle Cell Disease, run by National Institutes of Health Clinical Center (CC) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Oct 2018to 28 Aug 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH
Who may be able to join
- 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
Show all 9Show fewer
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
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.
Read more
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Keywords
- Informed Consent, Stem Cell Transplantation, Understanding, Optimism, Sickle Cell Disease
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| SCD patients | Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Expectations, understanding and motivations for enrollment
Understanding, expectations, motivations, and decision making process
Measured: Before or after enrollment in PBSCT or GT trial, but after decision made
Secondary outcomes (1)
Explore the role of family and religion
Description of the role of family, culture, and religion in enrollment decision making
Measured: After decision made about enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2018
- Study start29 Oct 2018
- Primary completion31 Aug 2019
- Study completion28 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Christine Grady, Ph.D., Principal investigator, National Institutes of Health Clinical Center (CC)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03709303
- Sponsor's protocol ID
- 190004
- Other IDs
- 19-CC-0004
- Status last verified
- Aug 2020
- First submitted
- 16 Oct 2018
- Last updated
- 1 Sep 2020
- Results posted
- No
Publications
Kim SY, De Vries R, Holloway RG, Kieburtz K. Understanding the 'therapeutic misconception' from the research participant's perspective. J Med Ethics. 2016 Aug;42(8):522-3. doi: 10.1136/medethics-2016-103597. Epub 2016 May 4. No abstract available.
PubMed 27145809Pentz RD, White M, Harvey RD, Farmer ZL, Liu Y, Lewis C, Dashevskaya O, Owonikoko T, Khuri FR. Therapeutic misconception, misestimation, and optimism in participants enrolled in phase 1 trials. Cancer. 2012 Sep 15;118(18):4571-8. doi: 10.1002/cncr.27397. Epub 2012 Jan 31.
PubMed 22294385Adegbola M. Spirituality, Self-Efficacy, and Quality of Life among Adults with Sickle Cell Disease. South Online J Nurs Res. 2011 Apr;11(1):5.
PubMed 21769284
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research
- ClinicalTrials.gov ID
- NCT03709303
- Acronym / sponsor ID
- 190004
- Conditions
- Sickle Cell Disease
- Keywords
- Informed Consent, Stem Cell Transplantation, Understanding, Optimism, Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- National Institutes of Health Clinical Center (CC)US National Institutes of Health
- Study start
- 29 Oct 2018 (actual)
- Primary completion
- 31 Aug 2019 (actual)
- Study completion
- 28 Aug 2020 (actual)
- First posted
- 17 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SCD patients | – | –Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH
Key inclusion criteria (9 listed)
- 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:
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
Explore the role of family and religion
Time frame: After decision made about enrollment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2020.
- Background: PubMed 27145809Kim SY, De Vries R, Holloway RG, Kieburtz K. Understanding the 'therapeutic misconception' from the research participant's perspective. J Med Ethics. 2016 Aug;42(8):522-3. doi: 10.1136/medethics-2016-103597. Epub 2016 May 4. No abstract available.
- Background: PubMed 22294385Pentz RD, White M, Harvey RD, Farmer ZL, Liu Y, Lewis C, Dashevskaya O, Owonikoko T, Khuri FR. Therapeutic misconception, misestimation, and optimism in participants enrolled in phase 1 trials. Cancer. 2012 Sep 15;118(18):4571-8. doi: 10.1002/cncr.27397. Epub 2012 Jan 31.
- Background: PubMed 21769284Adegbola M. Spirituality, Self-Efficacy, and Quality of Life among Adults with Sickle Cell Disease. South Online J Nurs Res. 2011 Apr;11(1):5.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christine Grady, Ph.D. principal investigator
National Institutes of Health Clinical Center (CC)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by National Institutes of Health Clinical Center (CC) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Expectations, understanding and motivations for enrollment (time frame: Before or after enrollment in PBSCT or GT trial, but after decision made). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03709303, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2020.