Sickle Cell DiseaseNCT03167450
Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care
An observational study of Sickle Cell Disease, run by National Human Genome Research Institute (NHGRI) at 1 site in United States.
Trial summary
Background: Sickle cell disease (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body and increase risk of blood clots. 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
- 109 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Apr 2017to 5 Feb 2021
- 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: Adults with sickle cell disease; parent of a child with sickle cell disease or physicians who have delivered healthcare to individuals living with sickle cell disease for at least a year
Who may be able to join
- The inclusion criteria for patients:
- must have a diagnosis of sickle cell disease
- must be 18 years or older
- must be English-speaking
- The inclusion criteria for parents of patients with SCD:
- must have a child with sickle cell disease
Show all 15Show fewer
- must be 18 years or older
- must be English speaking
- Lastly, the inclusion criteria for physicians:
- must care for sickle cell patients
- must have cared for sickle cell patients for a minimum of 12 months
- must have been the caregiver for at least five adult patients and/or five pediatric patients
- must be 18 years or older
- must be English-speaking.
- The participants need to be 18 years or older in order to provide informed consent. It is necessary that participants speak English due to the nature of the study. Because a moderated conversation will take place, it is essential that all participants and researchers speak the same language to allow for interactive discourse and comprehension.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Human Genome Research Institute (NHGRI)
Bethesda, Maryland, 20892
About this study
Background:
Sickle cell disease (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body and increase risk of blood clots. CRISPR Cas9 is a new tool which allows scientists to snip and edit genes in a way that is faster, cheaper, and more precise than other gene-editing tools. Recently, research has been done using CRISPR Cas9 to correct the sickle cell gene in animal models and human cells. Researchers want to understand the views of those with SCD, parents of people with SCD, and the providers of these patients regarding use of CRISPR Cas9 in clinical trials and treatment.
Read more
Objectives:
To study the attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing. An additional purpose of this study is to assess the utility of an educational tool for improving understanding of CRISPR Cas9.
Eligibility:
People ages 18 and older who speak English and either have SCD, are a parent of someone with SCD, or are a physician for people with SCD.
Design:
Participants will be screened via phone. Those with SCD will be screened with data from their SCD genotype.
Participation lasts about 2 hours.
Participants will fill out three surveys.
Participants will watch a video about CRISPR Cas9.
Participants will engage in a focus group session. This will be audiotaped and analyzed.
The data from the survey questions and focus groups may be used for future research. However, all personally identifiable information will be removed before data is shared.
Participants data will be identified with a code number instead of their name.
Participants may be invited to join future studies of SCD.
Read the detailed description
The CRISPR/Cas9 gene-editing technique is taking the scientific research and healthcare community by storm with the promise it holds to cure and ease the burden of debilitating diseases. However, there is limited knowledge regarding the implications of using this type of tool in human research and medicine. Researchers need to understand the viewpoints of patients, their families, and their providers, to ensure that the approach taken towards gene-editing is inclusive and respectful of different interests and concerns. The dialogue, thus far, has been dominated by scientific researchers, physician scientists, ethicists, public health and policy experts. It is important to the advancement of the science to study the patient s perspective about the use of the technology. In addition, parents often play important roles in the decision-making process; in this regard, understanding the views and questions of this group of individuals regarding CRISPR/Cas9 human use is essential. We conduct a qualitative study with a mixed methods component to investigate the knowledge, attitudes and beliefs of patients living with sickle cell disease (SCD), the parents of patients with SCD, and the physicians of this patient population regarding the use of CRISPR/Cas9 technology. The study is designed to measure these three cohorts baseline overall genetic literacy, CRISPR-specific literacy, and general attitudes and beliefs toward gene-editing/CRISPR Cas9(in both somatic and germline cells); to evaluate the utility of an educational tool in improving one s understanding of this innovative technique; and to gauge how attitudes and beliefs toward gene-editing, specifically CRISPR Cas9, perhaps shift or remain intact after the educational video, as well as within a focus group space.
- Conditions
- Sickle Cell Disease
- Keywords
- CRISPR, Gene Editing, Qualitative, Participation Clinical Trials, Genetic Literacy
- 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 |
|---|---|---|
| Adults | Adults have sickle cell disease | |
| Parents | Parents with children/adults who have sickle cell disease | |
| Physicians | Physicians who have delivered healthcare to individuals living with sickle cell disease for at least a year |
What is being measured
The primary outcome is the main question the study is designed to answer.
Focus group interviews related to attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing.
Measured: At the day of inclusion
To assess the utility of an educational tool for improving understanding of CRISPR Cas9.
Measured: At the day of inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2017
- Study start28 Apr 2017
- Primary completion31 Dec 2017
- Study completion5 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Family based
- Time perspective
- Cross-sectional
- Enrolment
- 109 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Vence L Bonham, J.D., Principal investigator, National Human Genome Research Institute (NHGRI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03167450
- Sponsor's protocol ID
- 999917081
- Other IDs
- 17-HG-N081
- Status last verified
- Feb 2021
- First submitted
- 24 May 2017
- Last updated
- 9 Feb 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care
- ClinicalTrials.gov ID
- NCT03167450
- Acronym / sponsor ID
- 999917081
- Conditions
- Sickle Cell Disease
- Keywords
- CRISPR, Gene Editing, Qualitative, Participation Clinical Trials, Genetic Literacy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 109 participants (actual)
- Lead sponsor
- National Human Genome Research Institute (NHGRI)US National Institutes of Health
- Study start
- 28 Apr 2017 (actual)
- Primary completion
- 31 Dec 2017 (actual)
- Study completion
- 5 Feb 2021 (actual)
- First posted
- 30 May 2017 (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 (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body and increase risk of blood clots. CRISPR Cas9 is a new tool which allows scientists to snip and edit genes in a way that is faster, cheaper, and more precise than other gene-editing tools. Recently, research has been done using CRISPR Cas9 to correct the sickle cell gene in animal models and human cells. Researchers want to understand the views of those with SCD, parents of people with SCD, and the providers of these patients regarding use of CRISPR Cas9 in clinical trials and treatment.
Objectives:
To study the attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing. An additional purpose of this study is to assess the utility of an educational tool for improving understanding of CRISPR Cas9.
Eligibility:
People ages 18 and older who speak English and either have SCD, are a parent of someone with SCD, or are a physician for people with SCD.
Design:
Participants will be screened via phone. Those with SCD will be screened with data from their SCD genotype.
Participation lasts about 2 hours.
Participants will fill out three surveys.
Participants will watch a video about CRISPR Cas9.
Participants will engage in a focus group session. This will be audiotaped and analyzed.
The data from the survey questions and focus groups may be used for future research. However, all personally identifiable information will be removed before data is shared.
Participants data will be identified with a code number instead of their name.
Participants may be invited to join future studies of SCD.
Detailed description
The CRISPR/Cas9 gene-editing technique is taking the scientific research and healthcare community by storm with the promise it holds to cure and ease the burden of debilitating diseases. However, there is limited knowledge regarding the implications of using this type of tool in human research and medicine. Researchers need to understand the viewpoints of patients, their families, and their providers, to ensure that the approach taken towards gene-editing is inclusive and respectful of different interests and concerns. The dialogue, thus far, has been dominated by scientific researchers, physician scientists, ethicists, public health and policy experts. It is important to the advancement of the science to study the patient s perspective about the use of the technology. In addition, parents often play important roles in the decision-making process; in this regard, understanding the views and questions of this group of individuals regarding CRISPR/Cas9 human use is essential. We conduct a qualitative study with a mixed methods component to investigate the knowledge, attitudes and beliefs of patients living with sickle cell disease (SCD), the parents of patients with SCD, and the physicians of this patient population regarding the use of CRISPR/Cas9 technology. The study is designed to measure these three cohorts baseline overall genetic literacy, CRISPR-specific literacy, and general attitudes and beliefs toward gene-editing/CRISPR Cas9(in both somatic and germline cells); to evaluate the utility of an educational tool in improving one s understanding of this innovative technique; and to gauge how attitudes and beliefs toward gene-editing, specifically CRISPR Cas9, perhaps shift or remain intact after the educational video, as well as within a focus group space.
Study design
- Observational model
- Family based
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Adults | – | –Adults have sickle cell disease |
| Parents | – | –Parents with children/adults who have sickle cell disease |
| Physicians | – | –Physicians who have delivered healthcare to individuals living with sickle cell disease for at least a year |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with sickle cell disease; parent of a child with sickle cell disease or physicians who have delivered healthcare to individuals living with sickle cell disease for at least a year
Key inclusion criteria (15 listed)
- must have a diagnosis of sickle cell disease
- must be 18 years or older
- must be English-speaking
- must have a child with sickle cell disease
- must be 18 years or older
Key exclusion criteria (0 listed)
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
Focus group interviews related to attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing.
Time frame: At the day of inclusion
To assess the utility of an educational tool for improving understanding of CRISPR Cas9.
Time frame: At the day of inclusion
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 9 Feb 2021.
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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.
Vence L Bonham, J.D. principal investigator
National Human Genome Research Institute (NHGRI)
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 Human Genome Research Institute (NHGRI) 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 15 inclusion and 0 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 9 Feb 2021. 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?
Focus group interviews related to attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing. (time frame: At the day of inclusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Feb 2021.