Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research

NCT03709303 | Completed

• 2 other identifiers: 19000419-CC-0004
• Study Type: observational
• Target: 27
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Sickle Cell Disease

Keywords

Informed Consent; Stem Cell Transplantation; Understanding; Optimism; Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Expectations, understanding and motivations for enrollment

  Understanding, expectations, motivations, and decision making process

  Before or after enrollment in PBSCT or GT trial, but after decision made

Secondary Outcomes (1)

• Explore the role of family and religion

  After decision made about enrollment

Study Arms (1)

SCD patients

Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy

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

Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• National Institutes of Health Clinical Center (CC): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• 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.

  PMID: 27145809 BACKGROUND

• Pentz 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.

  PMID: 22294385 BACKGROUND

• Adegbola M. Spirituality, Self-Efficacy, and Quality of Life among Adults with Sickle Cell Disease. South Online J Nurs Res. 2011 Apr;11(1):5.

  PMID: 21769284 BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

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

• Christine Grady, Ph.D.

  National Institutes of Health Clinical Center (CC)

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: October 16, 2018
• First Posted: October 17, 2018
• Study Start: October 29, 2018
• Primary Completion: August 31, 2019
• Study Completion: August 28, 2020
• Last Updated: September 1, 2020

Record last verified: 2020-08

Locations

US (1)