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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

This study is not yet open for participant recruitment.
Verified June 15, 2017 by National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
Sponsor:
ClinicalTrials.gov Identifier:
NCT03167450
First Posted: May 30, 2017
Last Update Posted: December 15, 2017
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.
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
  Purpose

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.


Condition
Sickle Cell Disease

Study Type: Observational
Study Design: Observational Model: Family-Based
Time Perspective: Cross-Sectional
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

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) ):

Primary Outcome Measures:
  • 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 ]

Estimated Enrollment: 90
Anticipated Study Start Date: December 20, 2017
Estimated Study Completion Date: June 30, 2018
Estimated Primary Completion Date: June 30, 2018 (Final data collection date for primary outcome measure)
Groups/Cohorts
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

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.
  Eligibility

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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
Criteria
  • INCLUSION CRITERIA:

The inclusion criteria for patients:

  1. must have a diagnosis of sickle cell disease
  2. must be 18 years or older
  3. must be English-speaking

The inclusion criteria for parents of patients with SCD:

  1. must have a child with sickle cell disease
  2. must be 18 years or older
  3. must be English speaking

Lastly, the inclusion criteria for physicians:

  1. must care for sickle cell patients
  2. must have cared for sickle cell patients for a minimum of 12 months
  3. must have been the caregiver for at least five adult patients and/or five pediatric patients
  4. must be 18 years or older
  5. 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.

  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT03167450


Contacts
Contact: Vence L Bonham, J.D. (301) 594-3973 bonhamv@nhgri.nih.gov

Locations
United States, Maryland
National Human Genome Research Institute (NHGRI) Not yet recruiting
Bethesda, Maryland, United States, 20892
Contact: Vence Bonham, J.D.    301-594-3973    bonhamv@nhgri.nih.gov   
Sponsors and Collaborators
National Human Genome Research Institute (NHGRI)
Investigators
Principal Investigator: Vence L Bonham, J.D. National Human Genome Research Institute (NHGRI)
  More Information

Responsible Party: National Human Genome Research Institute (NHGRI)
ClinicalTrials.gov Identifier: NCT03167450     History of Changes
Other Study ID Numbers: 999917081
17-HG-N081
First Submitted: May 24, 2017
First Posted: May 30, 2017
Last Update Posted: December 15, 2017
Last Verified: June 15, 2017

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) ):
CRISPR
Gene Editing
Qualitative
Participation Clinical Trials
Genetic Literacy

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn