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

NCT03167450 | Completed

• 2 other identifiers: 99991708117-HG-N081
• Study Type: observational
• Target: 109
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Sickle Cell Disease

Keywords

CRISPR; Gene Editing; Qualitative; Participation Clinical Trials; Genetic Literacy

Outcome Measures

Primary Outcomes (2)

• 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.

  At the day of inclusion

• To assess the utility of an educational tool for improving understanding of CRISPR Cas9.

  At the day of inclusion

Study Arms (3)

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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
• must be English speaking
• 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.

Sponsors & Collaborators

• National Human Genome Research Institute (NHGRI): lead

Study Sites (1)

National Human Genome Research Institute (NHGRI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Vence L Bonham, J.D.

  National Human Genome Research Institute (NHGRI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: FAMILY BASED
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2017
• First Posted: May 30, 2017
• Study Start: April 28, 2017
• Primary Completion: December 31, 2017
• Study Completion: February 5, 2021
• Last Updated: February 9, 2021

Record last verified: 2021-02

Locations

US (1)