Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

This study seeks to adapt Caregiver Speaks, a psychosocial supportive intervention delivered through Facebook, for parent caregivers of children living with Rett Syndrome. This study also seeks to explore feasibility and acceptability of implementing the adapted intervention, and its impact on parent psychological outcomes.

Study Overview

• Status: Not yet recruiting
• Conditions: Rett Syndrome; Social Support; Family Caregivers; Psychological Support
• Intervention / Treatment: Other: Those who have patients prescribed Daybue; Other: Caregiver Speaks + Daybue

Detailed Description

Rett syndrome (RTT) is a rare, progressive neurodevelopmental disorder characterized by early developmental regression, severe intellectual and physical disability, seizures, feeding and breathing abnormalities, and a markedly reduced lifespan. These complex medical needs require intensive, lifelong caregiving, most often provided by parents. As a result, RTT profoundly affects not only the child but the entire family system. Parent caregivers must navigate highly specialized healthcare systems while simultaneously providing continuous, around-the-clock care and managing significant emotional strain.

The diagnosis of RTT is frequently described as devastating for parents, who experience anticipatory grief, anxiety, parenting stress, and chronic distress as caregiving demands escalate across the lifespan. Making sense of the diagnosis-including the possibility of early death-can be especially challenging, as it contradicts deeply held expectations that parents will outlive their children. Research in RTT and other rare pediatric conditions demonstrates that caregiver psychological distress is strongly linked to caregiving burden, treatment adherence, and child quality of life, highlighting the inseparability of patient and family wellbeing.

Despite this, RTT care has historically focused almost exclusively on medical management, with limited availability of evidence-based psychosocial interventions tailored to the unique emotional and social experiences of RTT caregivers. There is a critical need for innovative, scalable approaches that support caregiver wellbeing, reduce isolation, and integrate meaning-centered psychosocial care alongside pharmacologic treatment.

Overview of the Intervention This project centers on adapting and evaluating Caregiver Speaks, a meaning-centered, storytelling-based psychosocial intervention originally developed for family caregivers of individuals with dementia and cancer. Caregiver Speaks is grounded in Park and Folkman's meaning-making model of stress and coping, which posits that individuals adapt to stressful life events by reconstructing and integrating new meaning into their personal narratives.

The intervention uses guided photo-elicitation and moderated online group discussion to help caregivers reflect on their caregiving experiences, reframe sources of distress, and build emotional connection with peers facing similar challenges. Prior studies demonstrate that Caregiver Speaks improves caregiver psychological outcomes by reducing stress and grief intensity while enhancing meaning, purpose, and social support.

However, Caregiver Speaks has never been adapted for caregivers of children with life-limiting neurodevelopmental conditions such as RTT. Preliminary research with RTT families indicates that the intervention requires significant adaptation to address the medical complexity, prolonged caregiving trajectory, and profound social isolation experienced by RTT caregivers. This study represents the first effort to optimize Caregiver Speaks for the RTT community and integrate it with emerging pharmacologic treatments.

Study Objectives and Aims The overarching goal of this project is to enhance whole-family wellbeing in RTT by developing, piloting, and preliminarily evaluating Caregiver Speaks-RTT, an adapted version of the intervention designed specifically for caregivers of individuals with RTT.

Aim 1: Adapt the Caregiver Speaks intervention for RTT caregivers using Rapid Community Translation (RCT) methodology.

Parents of children with RTT and clinicians from RTT Centers of Excellence (CoE) will collaboratively refine intervention content, format, and delivery to ensure relevance, acceptability, and clinical feasibility while preserving the intervention's core components.

Aim 2: Evaluate the feasibility and acceptability of Caregiver Speaks-RTT through a single-arm, mixed-methods pilot study.

Two sequential cohorts of caregivers will participate in the 6-week online intervention, allowing for iterative refinement between cohorts. Quantitative and qualitative data will assess feasibility, engagement, retention, and caregiver perceptions of acceptability and appropriateness.

Aim 3: Conduct an exploratory randomized comparative study to examine caregiver outcomes when Caregiver Speaks-RTT is integrated with pharmacologic treatment.

Caregivers will be randomized to receive either pharmacologic treatment alone or pharmacologic treatment plus Caregiver Speaks-RTT, allowing for preliminary exploration of additive benefits to caregiver wellbeing, family functioning, and medication adherence.

Study Design and Methods This is a multi-phase, mixed-methods study conducted over two years. Aim 1: Rapid Community Translation (Adaptation Phase) A working group of approximately 10 participants-including RTT caregivers and CoE clinicians-will engage in a series of structured, virtual sessions facilitated by an interdisciplinary research team with expertise in social work, psychology, neurology, rare disease research, and qualitative methods. Sessions will include education on caregiver distress in RTT, discussion of meaning-making and social support, and hands-on review of Caregiver Speaks materials. Participant feedback will guide systematic adaptations to ensure cultural relevance, clarity, and feasibility for RTT families and clinical care environments.

Aim 2: Feasibility and Acceptability Pilot Twenty caregivers of individuals with RTT will participate in two cohorts of the adapted Caregiver Speaks-RTT intervention. Each cohort will engage in a 6-week, asynchronous, moderated private Facebook group. Caregivers will share photos and stories reflecting their caregiving experiences, respond to weekly prompts, and engage with peers.

Feasibility will be assessed through recruitment, retention, session participation, and completeness of outcome data. Acceptability and appropriateness will be evaluated using validated surveys and post-intervention qualitative interviews. Interviews will also explore caregivers' experiences with medication adherence, including perceived barriers and facilitators, to inform integrated care strategies.

Aim 3: Exploratory Comparative Trial Thirty caregivers will be randomized into two groups: pharmacologic treatment plus Caregiver Speaks-RTT, or pharmacologic treatment alone (with delayed access to the intervention). Outcomes will be measured at baseline and post-intervention using validated scales assessing caregiver mental health, caregiving burden, parenting stress, meaning and purpose, social isolation, medication barriers, and child quality of life. Although not powered for efficacy, this exploratory analysis will generate critical proof-of-concept data.

Data Analysis Quantitative data will be analyzed using descriptive statistics and exploratory comparisons to examine feasibility benchmarks, acceptability thresholds, and pre-post changes in outcomes. Qualitative interview data will be analyzed using grounded theory and convergent mixed-methods approaches to contextualize quantitative findings and identify mechanisms of change. Adaptations will be documented using established reporting frameworks to support reproducibility and future dissemination.

Ethical Considerations This study involves minimal risk. Participation may evoke emotional responses related to sensitive caregiving experiences; all intervention activities are optional, and participants may withdraw at any time. Confidentiality is protected through secure data systems, private online groups, and de-identification procedures. All procedures are reviewed and approved by the Colorado Multiple Institutional Review Board.

Impact and Future Directions This study will generate critical preliminary evidence for an innovative, integrated model of care that addresses both the medical and psychosocial needs of families affected by RTT. By supporting caregiver meaning-making, reducing distress, and strengthening peer support, Caregiver Speaks-RTT has the potential to improve family functioning, enhance treatment adherence, and positively influence child quality of life.

Findings will directly inform a future, fully powered NIH-funded clinical trial and guide broader implementation of Caregiver Speaks-RTT across RTT Centers of Excellence. More broadly, this work establishes a scalable framework for integrating psychosocial support with pharmacologic treatment in rare pediatric disorders, advancing whole-family care and community health.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Abigail Rolbiecki, PhD, MPH, MSW; Phone Number: 970-491-6204; Email: abigail.rolbiecki@colostate.edu
• Study Contact Backup: Name: Talia Thompson, PhD; Phone Number: 303-871-3736

Study Locations

• United States
  • Colorado
    • Fort Collins, Colorado, United States, 80523
      • Colorado State University
      • Contact: Abigail Rolbiecki, PhD, MPH, MSW; Phone Number: 970-491-6204; Email: abigail.rolbiecki@colostate.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adult caregiver (≥18 years old) of an individual with Rett syndrome Caregiver of a child or individual of any age with a confirmed clinical diagnosis of Rett syndrome.
• English-speaking and able to read and understand study materials Willing and able to participate in a 6-week, online psychosocial intervention delivered via a private social media platform
• Willing and able to complete electronic surveys and, if selected, participate in virtual interviews
• For randomized phase participation: child is receiving standard pharmacologic treatment for Rett syndrome (e.g., Daybue) as part of routine clinical care

Exclusion Criteria:

• Inability to speak, read, or understand English sufficiently to participate in the intervention and assessments.
• Caregiver under the age of 18 years
• Lack of confirmed diagnosis of Rett syndrome in the care recipient.
• Inability or unwillingness to provide informed consent.
• Lack of access to required technology (internet-enabled device and ability to access the online intervention platform).

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Pharmacologic Treatment Only; Participants in this arm will receive standard pharmacologic treatment for Rett syndrome (e.g., Daybue) as prescribed by their clinical care team. Participants in this arm will not receive the Caregiver Speaks-RTT psychosocial intervention during the randomized study period but will be offered the opportunity to participate in the Caregiver Speaks-RTT intervention after completion of study follow-up assessments (wait-list control).	Other: Those who have patients prescribed Daybue; Participants must be receiving Daybue (trofinetide) as prescribed and managed by their treating clinician as part of routine clinical care. The study does not assign, initiate, modify, or manage pharmacologic treatment. Participants are required to continue taking Daybue according to their physician's prescription during the study period. Medication dosing, monitoring, and clinical decision-making remain entirely under the care of the participant's healthcare provider.
Experimental: Pharmacologic Treatment Plus Caregiver Speaks-RTT; Participants in this arm will receive standard pharmacologic treatment for Rett syndrome (e.g., Daybue) in combination with the Caregiver Speaks-RTT psychosocial intervention, a 6-week, moderated, online, meaning-centered storytelling program designed to support caregiver wellbeing.	Other: Those who have patients prescribed Daybue; Participants must be receiving Daybue (trofinetide) as prescribed and managed by their treating clinician as part of routine clinical care. The study does not assign, initiate, modify, or manage pharmacologic treatment. Participants are required to continue taking Daybue according to their physician's prescription during the study period. Medication dosing, monitoring, and clinical decision-making remain entirely under the care of the participant's healthcare provider.; Other: Caregiver Speaks + Daybue; Participants receive standard pharmacologic treatment for Rett syndrome (e.g., Daybue [trofinetide]) as prescribed and managed by their clinical care team. Medication use follows routine clinical practice, including dosing, monitoring, and management of side effects. No experimental medication dosing or alteration is required as part of this study. Pharmacologic treatment is provided either alone or in combination with the Caregiver Speaks-RTT psychosocial intervention, depending on randomized group assignment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility of Intervention Measure (FIM)	Feasibility of the Caregiver Speaks-RTT intervention assessed by caregiver recruitment, retention, session participation, and completeness of outcome data. Feasibility benchmarks include ≥80% intervention engagement, ≥80% retention through completion, and <20% missing baseline or post-intervention data, measured using the Feasibility of Intervention Measure (FIM) and participation metrics. 1 to 5 (average score across 4 items; alternatively 4 to 20 if summed).	Immediately post-intervention (6 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acceptability of Intervention Measure (AIM)	Caregiver-reported acceptability and perceived appropriateness of the Caregiver Speaks-RTT psychosocial intervention, assessed using the Acceptability of Intervention Measure (AIM). Measures assess satisfaction with intervention content, delivery format, relevance to caregiving for individuals with Rett syndrome, and perceived fit within caregivers' needs. 1 to 5 (average score across 4 items; alternatively 4 to 20 if summed).	Immediately post-intervention (6 weeks)
Intervention Appropriateness Measure (IAM)	Caregiver-reported perceived appropriateness of the Caregiver Speaks-RTT intervention, assessed using the Intervention Appropriateness Measure (IAM). This measure evaluates perceived fit, relevance, and suitability of the intervention for caregivers of individuals with Rett syndrome. 1 to 5 (average score across 4 items; alternatively 4 to 20 if summed).	Immediately post-intervention (6 weeks).

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Interviews to Qualitatively Assess Acceptability and Implementation Feedback	Qualitative assessment of caregiver experiences with Caregiver Speaks-RTT obtained through semi-structured post-intervention interviews. Interviews explore perceived benefits, challenges, barriers and facilitators to participation, and suggestions for intervention refinement and clinical integration.	Immediately post-intervention (6-weeks).
Intervention Engagement and Participation	Intervention engagement assessed by session participation metrics, including proportion of weekly prompts responded to and overall participation across the 6-week intervention period. Engagement thresholds are used to support feasibility and acceptability assessments.	Weekly during the 6-week intervention period.
Parenting Stress Index-Short Form	Caregiver-reported stress and caregiving burden assessed using the Parenting Stress Index-Short Form and the RTT Caregiver Inventory to explore changes associated with pharmacologic treatment alone versus pharmacologic treatment plus Caregiver Speaks-RTT. Minimum score is 36 to maximum 180. Higher scores indicate greater parenting stress (worse outcome). The PSI-SF contains 36 items scored 1-5.	Baseline and post-intervention (6 weeks)
Medication Adherence and Barriers	Caregiver-reported barriers and facilitators to medication adherence assessed using the Parent Medication Barriers Scale and qualitative interview questions to contextualize pharmacologic treatment experiences.	Baseline and post-intervention (6 weeks)
Quality of Life Inventory-Disability (QI-D)	Parent-proxy report of child quality of life and clinical functioning assessed using the Quality of Life Inventory-Disability (QI-D). 32 to 160 raw score (32 items scored 1-5); often transformed to 0-100 domain scores. Higher scores indicate better quality of life (better outcome).	Baseline and post intervention
NIH PROMIS Anxiety Measure	Caregiver-reported anxiety assessed using NIH PROMIS Anxiety measures to explore changes following participation in pharmacologic treatment alone or pharmacologic treatment plus the Caregiver Speaks-RTT psychosocial intervention. Standardized T-score, approximately 0-100, population mean = 50, SD = 10. Higher scores indicate more anxiety symptoms (worse outcome).	Baseline and post-intervention
NIH PROMIS Depression Measure	Caregiver-reported depressive symptoms assessed using NIH PROMIS Depression measures to explore changes following pharmacologic treatment alone versus pharmacologic treatment combined with the Caregiver Speaks-RTT intervention. Standardized T-score, approximately 0-100, population mean = 50, SD = 10. Higher scores indicate more depressive symptoms (worse outcome).	Baseline and post-intervention
NIH PROMIS Emotional Distress Measure	Caregiver-reported emotional distress assessed using NIH PROMIS Emotional Distress measures to examine changes associated with participation in pharmacologic treatment alone or pharmacologic treatment plus Caregiver Speaks-RTT. Standardized T-score, approximately 0-100, population mean = 50, SD = 10. Higher scores indicate more emotional distress (worse outcome).	Baseline and post-intervention
NIH PROMIS Global Health Measure	Caregiver-reported overall mental health assessed using NIH PROMIS Global Health measures to explore changes following pharmacologic treatment alone or pharmacologic treatment in combination with the Caregiver Speaks-RTT intervention. Standardized T-score, approximately 0-100, population mean = 50, SD = 10. Higher scores indicate better overall health and well-being (better outcome).	Baseline and post-intervention

Collaborators and Investigators

• Sponsor: Colorado State University
• Collaborators: Children's Hospital Colorado; University of Denver

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): May 31, 2028

Study Registration Dates

• First Submitted: June 5, 2026
• First Submitted That Met QC Criteria: July 22, 2026
• First Posted (Actual): July 28, 2026

Study Record Updates

• Last Update Posted (Actual): July 28, 2026
• Last Update Submitted That Met QC Criteria: July 22, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Family Caregivers; Rett Syndrome; Online Support Group; Meaning-making
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, Inborn; Neurobehavioral Manifestations; Heredodegenerative Disorders, Nervous System; Intellectual Disability; Genetic Diseases, X-Linked; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; X-Linked Intellectual Disability; Rett Syndrome; trofinetide
• Other Study ID Numbers: 7932; IIS-RS-2025-006 (Other Grant/Funding Number: Acadia Pharmaceuticals)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: De-identified individual participant data (IPD) underlying published results may be shared with qualified researchers upon reasonable request and approval by the study investigators. Shared data will exclude direct identifiers and will be limited to quantitative survey data; qualitative interview transcripts will not be shared due to the risk of re-identification. Data sharing will be subject to data use agreements and ethical approvals, as appropriate.
• IPD Sharing Time Frame: De-identified individual participant data (IPD) and supporting documentation will be available beginning after publication of the primary study results, anticipated within 12 months of study completion. Data will remain available for a minimum of 5 years following publication, subject to continued data stewardship and institutional policies.
• IPD Sharing Access Criteria: De-identified IPD and supporting information will be available to qualified researchers for research purposes only. Data requests must include a brief proposal outlining the intended use of the data and must receive approval from the study investigators. Shared IPD will be limited to quantitative survey and assessment data; qualitative interview transcripts and audio recordings will not be shared due to the risk of participant re-identification. Access will be granted through secure data transfer following execution of an appropriate data use agreement and confirmation of ethical approvals where applicable.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; ANALYTIC_CODE

Drug and device information, study documents

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