Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA) (EMPOWER-AYA)

This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 60 AYAs with SCD ages 15-25 and their caregivers and 8 SCD providers will participate in the pilot pragmatic trial. AYAs, caregivers, and providers will be recruited from Nemours Children's Hospital, Delaware (NCH-DE), Nemours Children's Hospital in Orlando, FL (NCH-ORL), and Nemours Children's Health at Wolfson Children's Hospital in Jacksonville, FL (NCH-JAX). NCH-DE participants (n=30) will receive the SDM intervention including a virtual reality patient health education component, whereas NCH-ORL and NCH-JAX participants (n=30) will receive the SDM intervention with standard patient education materials (print, video). SCD providers will be trained to use the toolkit components and will introduce decision aids during an outpatient clinic visit for AYAs who are candidates for one or more disease-modifying therapies.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Behavioral: SCD Shared Decision-Making Toolkit for AYAs - including VR; Behavioral: SCD Shared Decision-Making Toolkit for AYAs - without VR

• Study Type: Interventional
• Enrollment (Estimated): 68
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Clinical Research Coordinator; Phone Number: 302-723-9331; Email: benjamin.bear@nemours.org

Study Locations

• United States
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Recruiting
      • Nemours Children's Hospital, Delaware

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients diagnosed with SCD ages 15-25 years receiving treatment at Nemours and their caregivers will be included. Patients must be deemed candidates for initiation of one or more disease-modifying therapies by their SCD healthcare provider.
• Nemours SCD healthcare providers will also be included.
• English language fluency is a requirement for all participants.

Exclusion Criteria:

• Patients/ caregivers who previously participated in usability testing of the intervention will be excluded.
• Patients will also be excluded if they have history of seizures, if their medical status or cognitive functioning preclude completion of interviews, or if no legal guardian is available to provide consent (for patients < 18 years).
• Providers still completing training will be excluded.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Shared Decision Making Toolkit including VR	Behavioral: SCD Shared Decision-Making Toolkit for AYAs - including VR; The SCD Shared Decision-Making Toolkit for AYAs (SDMT-AYA) targets multi-level barriers to uptake of disease-modifying therapies by providing technology-enhanced tools for: 1) clinicians (i.e., training in motivational interviewing, values elicitation, and cultural humility); 2) AYAs and caregivers (i.e., user-centered multimedia decision aids, including virtual reality, online (videos, animations, articles), and print materials); and 3) implementation (e.g., clinic audit and feedback strategies).
Experimental: Shared Decision Making Toolkit without VR	Behavioral: SCD Shared Decision-Making Toolkit for AYAs - without VR; The SCD Shared Decision-Making Toolkit for AYAs (SDMT-AYA) targets multi-level barriers to uptake of disease-modifying therapies by providing technology-enhanced tools for: 1) clinicians (i.e., training in motivational interviewing, values elicitation, and cultural humility); 2) AYAs and caregivers (i.e., user-centered multimedia decision aids, including online videos, animations, and articles as well as print materials); and 3) implementation (e.g., clinic audit and feedback strategies).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acceptability of Intervention Measure (AIM)	Questionnaire assessing healthcare provider perspectives on acceptability of the intervention. Total score ranges from 4-20, with higher scores indicating greater acceptability.	Within 2 weeks post-intervention
Patient/Caregiver Satisfaction Questionnaire	Survey assessing patient/caregiver satisfaction with the intervention. Survey will be developed by the research team, and additional details will be included here once items and scoring are finalized.	Within 2 weeks post-intervention
Feasibility of Intervention Measure (FIM)	Questionnaire assessing healthcare provider perspectives on the feasibility of the intervention. Total score ranges from 4-20, with higher scores indicating greater feasibility.	Within 2 weeks post-intervention
Participant Recruitment Rate	Rate of participant enrollment and retention in the study	Time of enrollment through study completion, approximately 12 months
Shared Decision Making Questionnaire (SDM-Q-9)	Questionnaire assessing patient- and caregiver-reported engagement in decision making. Total score ranges from 0 to 100, with higher scores indicating greater engagement in decision making.	Within 2 weeks post-intervention
Disease-Modifying Therapy Knowledge Questionnaire	Questionnaire assessing patient and caregiver knowledge of disease modifying-therapies for sickle cell disease. Survey will be developed by the research team, and additional details will be included here once items and scoring are finalized.	Within 2 weeks post-intervention

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Initiation of disease-modifying therapy	Proportion of patients who agree to initiate a disease-modifying therapy following use of the shared decision making intervention	Within 6 months post-intervention

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acute healthcare utilization	ED visits and hospitalizations for sickle cell-related complications during 6 months following the intervention	Within 6 months post-intervention

Collaborators and Investigators

• Sponsor: Nemours Children's Clinic

Study record dates

Study Major Dates

• Study Start (Actual): November 26, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: May 9, 2022
• First Submitted That Met QC Criteria: May 17, 2022
• First Posted (Actual): May 20, 2022

Study Record Updates

• Last Update Posted (Actual): June 16, 2026
• Last Update Submitted That Met QC Criteria: June 15, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell
• Other Study ID Numbers: 348507

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No