Decisional Capacity and Informed Consent in Fragile X Syndrome

• ClinicalTrials.gov Identifier: NCT02465931
• Recruitment Status: Completed
• First Posted: June 9, 2015
• Results First Posted: December 6, 2018
• Last Update Posted: March 7, 2019
• Sponsor: RTI International
• Collaborator: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Information provided by (Responsible Party): RTI International

Tracking Information

• First Submitted Date: May 22, 2015
• First Posted Date: June 9, 2015
• Results First Submitted Date: November 12, 2018
• Results First Posted Date: December 6, 2018
• Last Update Posted Date: March 7, 2019
• Actual Study Start Date: March 14, 2016
• Actual Primary Completion Date: March 21, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 25, 2019)

Decisional Capacity - Understanding Score [ Time Frame: Day 1, immediately following presentation of the material in the intervention or comparison condition ]

MacArthur Decisional Capacity - understanding score, range 0 (no understanding) to 26 (perfect understanding) A summary score was calculated, ranging from 0-26. Scores were then translated into a percentage based on the number of questions answered. A score of 100% indicates that the participant answered each understanding question correctly on the first attempt.

Original Primary Outcome Measures (submitted: June 4, 2015)

Decisional capacity assessment [ Time Frame: Up to 12 months ]

This will only include individuals who took part in the Aim 1 assessments. The sample size will be approximately 75 or more; however, eligibility and travel will play a role in how many we can feasibly conduct. Siblings will be eligible to participate so long as testing is done separately. The Aim 1 team members will provide an estimate of decisional capacity based on an individual's performance on the MacCAT flip chart and in consideration of the other assessments. Those in the first 3 categories will be eligible for the randomized control trial but those in the fourth category will not.

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Decisional Capacity and Informed Consent in Fragile X Syndrome
• Official Title: Decisional Capacity and Informed Consent in Fragile X Syndrome
• Brief Summary: The Decisional Capacity and Informed Consent in Fragile X Syndrome (FXS) project is for the Eunice Kennedy Shriver National Institute of Child Health & Human Development, 1R01HD071987-01A1, and will provide the first comprehensive description of decisional capacity of individuals with FXS; identify individual, family, and experiential factors associated with variability in decisional capacity; determine the validity of caregiver and expert ratings; and develop evidence-based guidelines for categorizing decisional capacity of individuals with FXS. The researchers will develop a methodologically rigorous and conceptually grounded decision aid using digital technology to enhance participation of individuals with FXS in the consent process.
• Detailed Description: FXS is the most common inherited form of intellectual disability (ID). The typical male has an IQ (intelligence quotient) of 50 to 55, but involvement ranges from mild to severe. Females are generally less affected and have a broader range in function. Some have severe ID and adaptive impairments, but most have a normal or borderline intellectual ability. This wide range in cognitive skills leads to variable ability to make choices and live independently. Most research on individuals with FXS has been noninvasive, limited to parent surveys and psychological assessments. Studies such as these evoke few if any major ethical concerns, so the consent process typically involves a straightforward parental consent. Some studies have drawn blood, collected saliva, measured heart rate, or used neuroimaging, but until recently there has been a paucity of treatment research. This scenario has changed dramatically in the past several years. Advances in understanding the molecular basis of FXS have led to a new generation of treatments, and clinical trials are under way using a variety of compounds. The possibility of side effects and the potential for significant changes in behavior and ability elevate to a new level the importance of obtaining meaningful consent, not only from parents, but also from individuals with FXS. Researchers and Institutional Review Board (IRB) members need data to guide decisions about involving individuals with FXS in the consent process. Unfortunately, little is known about the extent to which individuals with FXS can be or are involved in decisions about research participation. This project will assess the range of decisional capacity in FXS, identify factors associated with individual differences in decisional capacity, validate a rating scale for categorizing decisional capacity, and develop a novel tablet-based decision aid technology to assist researchers and clinicians in maximizing decisional capacity and consent. This study will work toward achieving two overarching aims: 1) Characterize and explain individual differences in decisional capacity in FXS, 2) Design and evaluate the effect of a tablet-based decision aid on participation in the consent process for a hypothetical clinical trial. Ultimately, this study will provide important information about the nature of decisional capacity in FXS and validate a novel intervention to maximize successful participation in the consent process.
• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Screening
• Condition: Fragile X Syndrome (FXS)

Intervention

• Other: Paper and Pencil Informed Consent
  Paper-based informed consent form will be sent to participant and family before data collection visit. They will be able to review as many times as they wish before visit. During visits, simplified overview of informed consent form will be provided in person just once. For MacArthur Competence Assessment Tool (MacCAT) Questions, All questions will be asked after the disclosure information has been presented. Questions will have the same wording as experimental condition. Procedures will mimic MacCAT/flipchart (incorrect or partial credit will be given opportunity to answer question again after disclosure information is repeated). There will be multiple choice options rather than open-ended. Finally, data collection will be done through paper and pencil.
• Other: Digital Informed Consent Tool
  Paper informed consent form will be sent to participant and family before data collection visit. They will be able to review as many times as they wish before visit. During visits, the participant can go through the tablet-based tool up to 3 times. All questions will be embedded within the vignettes/presentation of disclosure information. Question will use simplified wording, similar to flipchart. Procedures will mimic MacArthur Competence Assessment Tool (MacCAT) /flipchart (incorrect or partial credit will be given opportunity to answer question again after disclosure information is repeated). Multiple choice options will be utilized rather than open-ended. Finally, response data stored within tool and exported to dataset for analysis.

Study Arms

• Comparison Condition
  Paper and pencil informed consent
  Intervention: Other: Paper and Pencil Informed Consent
• Experimental: Intervention condition
  Digital informed consent tool
  Intervention: Other: Digital Informed Consent Tool

Publications *

• Furberg RD, Raspa M, Wheeler AC, McCormack LA, Bailey DB. A Digital Health App to Assess Decisional Capacity to Provide Informed Consent: Protocol for a Randomized Controlled Trial. JMIR Res Protoc. 2018 Nov 19;7(11):e10360. doi: 10.2196/10360.
• Furberg RD, Ortiz AM, Moultrie RR, Raspa M, Wheeler AC, McCormack LA, Bailey DB Jr. A Digital Decision Support Tool to Enhance Decisional Capacity for Clinical Trial Consent: Design and Development. JMIR Res Protoc. 2018 Jun 6;7(6):e10525. doi: 10.2196/10525.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 7, 2019): 152
• Original Estimated Enrollment (submitted: June 4, 2015): 200
• Actual Study Completion Date: March 21, 2017
• Actual Primary Completion Date: March 21, 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Places in the first 3 categories of the MacCAT flip chart
• Can provide informed consent using a standard practice

Exclusion Criteria:

• Not in the first 3 categories of the MacCAT flipchart
• Not able to provide informed consent without significant modifications to consent procedures

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 40 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02465931
• Other Study ID Numbers: FXS; 1R01HD071987-01A1 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: RTI International
• Original Responsible Party: Same as current
• Current Study Sponsor: RTI International
• Original Study Sponsor: Same as current
• Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Investigators

• Study Director: Robert Furberg, PhD; RTI International

• PRS Account: RTI International
• Verification Date: February 2019