Ignite Creation Date:
2025-12-24 @ 7:48 PM
Ignite Modification Date:
2025-12-30 @ 6:01 PM
Study NCT ID:
NCT03709303
Status:
COMPLETED
Last Update Posted:
2020-09-01
First Post:
2018-10-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-10-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-08', 'completionDateStruct': {'date': '2020-08-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-08-28', 'studyFirstSubmitDate': '2018-10-16', 'studyFirstSubmitQcDate': '2018-10-16', 'lastUpdatePostDateStruct': {'date': '2020-09-01', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-10-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-08-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Expectations, understanding and motivations for enrollment', 'timeFrame': 'Before or after enrollment in PBSCT or GT trial, but after decision made', 'description': 'Understanding, expectations, motivations, and decision making process'}], 'secondaryOutcomes': [{'measure': 'Explore the role of family and religion', 'timeFrame': 'After decision made about enrollment', 'description': 'Description of the role of family, culture, and religion in enrollment decision making'}]}, 'conditionsModule': {'keywords': ['Informed Consent', 'Stem Cell Transplantation', 'Understanding', 'Optimism', 'Sickle Cell Disease'], 'conditions': ['Sickle Cell Disease']}, 'referencesModule': {'references': [{'pmid': '27145809', 'type': 'BACKGROUND', 'citation': "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': '22294385', 'type': 'BACKGROUND', 'citation': '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': '21769284', 'type': 'BACKGROUND', 'citation': '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.'}], 'seeAlsoLinks': [{'url': 'https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2019-CC-0004.html', 'label': 'NIH Clinical Center Detailed Web Page'}]}, 'descriptionModule': {'briefSummary': 'Background:\n\nSickle 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.\n\nObjective:\n\nTo learn about the motivations, decisions, and experiences in clinical research of people with sickle cell disease.\n\nEligibility:\n\nAdults 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.\n\nDesign:\n\nParticipants 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.\n\nThe interview will be audio-recorded if the participant agrees.\n\nParticipants will be asked about:\n\n* Their experiences with and thoughts on sickle cell disease\n* Their decision to participate in clinical research\n* Factors that may have affected their decision to participate. These may include family, disease history, or faith.\n\nParticipants may complete a few brief questionnaires.', 'detailedDescription': '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.\n\nThis 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.\n\nPrimary Hypothesis:\n\nThis is a descriptive, explorative study. It may generate hypotheses for future studies.\n\nPurpose of the Study Protocol:\n\nTo 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Sample will be selected from among SCD patients enrolled in NHLBI protocols at NIH', 'healthyVolunteers': False, 'eligibilityCriteria': '* INCLUSION CRITERIA:\n\nOur inclusion criteria for study subjects are:\n\n* Adult (18+ years and older)\n* 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:\n\n * Screening study: 08-H-0156\n * Natural history protocol: 04-H-0161\n * PBSCT: 09-H-0225, 17-H-0069, 14-H-0077, 03-H-0170\n * GT: 14-H-0155\n* 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)\n* 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.\n\nEXCLUSION CRITERIA:\n\nStudy subjects will be excluded if they:\n\n* Lack cognitive capacity\n* Are not English speaking\n* Have not made a decision about participation in one of the GT or PBSCT protocols for which they are eligible'}, 'identificationModule': {'nctId': 'NCT03709303', 'briefTitle': 'Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research', 'organization': {'class': 'NIH', 'fullName': 'National Institutes of Health Clinical Center (CC)'}, 'officialTitle': 'Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research', 'orgStudyIdInfo': {'id': '190004'}, 'secondaryIdInfos': [{'id': '19-CC-0004'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'SCD patients', 'description': 'Patients with sickle cell disease who have decided about enrollment in an NIH study of PBSCT or Gene therapy'}]}, 'contactsLocationsModule': {'locations': [{'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'National Institutes of Health Clinical Center', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}], 'overallOfficials': [{'name': 'Christine Grady, Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Institutes of Health Clinical Center (CC)'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Institutes of Health Clinical Center (CC)', 'class': 'NIH'}, 'responsibleParty': {'type': 'SPONSOR'}}}}