NCT05999448

Brief Summary

The goal of this clinical trial is to learn which types of telehealth-based treatments best fit the needs of caregivers of people with rare neurogenetic conditions. The main questions it plans to answer are:

  • Which telehealth support programs best meet the needs of rare disorder caregivers?
  • How can individuals be matched to support programs that are right for them? What aspects of an individual (e.g., demographics, mental health symptoms, family characteristics, lifestyle) predict whether treatment will be a good fit?
  • Does peer-to-peer coaching help improve patients' experiences during telehealth treatment? Participants will be asked to complete a 12-week treatment program, which may include self-guided resources, individual therapies, group therapies, and/or peer-to-peer coaching. Before, during, and after treatment, participants will complete questionnaires to help researchers understand their experiences, symptoms, and impressions of their support program. Questionnaires will include both standard forms (administered up to 5 times throughout the study) and brief "snapshot surveys" that participants complete on their smartphones up to 3 times per day. Some participants will be assigned to a waitlist control, which means that they will provide data while they are not yet completing a support program. These participants will be assigned to a support program in the next treatment phase.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
800

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Jun 2023

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 28, 2023

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

July 17, 2023

Completed
1 month until next milestone

First Posted

Study publicly available on registry

August 21, 2023

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 8, 2025

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 29, 2025

Completed
Last Updated

August 21, 2023

Status Verified

August 1, 2023

Enrollment Period

2.2 years

First QC Date

July 17, 2023

Last Update Submit

August 11, 2023

Conditions

Outcome Measures

Primary Outcomes (14)

  • Change from Baseline on DASS-21 Total-Score at End-of-Treatment Survey

    The patient-reported Depression, Anxiety, and Stress Scale, 21 Total Score ranges from 0-63; higher scores indicate greater impairment. Change is measured as (Week 12-Baseline), statistically controlling for Baseline levels.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on PSOC Total Score at End-of-Treatment Survey

    The patient-reported Parenting Sense of Competence Total Score includes ranges from 17-102; higher scores indicate greater parenting sense of competency. Wording was adapted from the original 1978 measure to be inclusive of caregivers who are not biological parents or mothers. Change is measured as (Week 12-Baseline), statistically controlling for Baseline symptoms.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on PSI-4-SF Total Score at End-of-Treatment Survey

    The patient-reported Parenting Stress Index, 4th Edition (PSI-4-SF) Total T-Score ranges from 0-100; higher scores indicate greater stress. Change is measured as (Week 12-Baseline), statistically controlling for Baseline symptoms.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change in Average PANAS Negative Affect between Baseline and End-of-Treatment

    The patient-reported Positive and Negative Affect Scale (10 items) Negative Affect subscale total score ranges from 10-50; higher scores indicate greater negative affect. For each evaluation period, scores will be averaged across week-long patient-reported ecological momentary assessments, which are collected 3 times per day and 7 days per week. Change will be measured as (Week 12-Baseline), statistically controlling for Baseline levels.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change in Average Momentary Stress between Baseline and End-of-Treatment

    The patient-reported momentary stress item ranges from 0-100; higher scores indicate greater stress. For each evaluation period, scores will be averaged across week-long patient-reported ecological momentary assessments, which are collected 3 times per day and 7 days per week. Change will be measured as (Week 12-Baseline), statistically controlling for Baseline levels.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on CGI-S at End-of-Treatment

    The Clinical Global Impression-Severity (CGI-S) is rated by the patient's primary clinician on a scale of 1-7, with higher scores indicating greater impairment. Change will be measured as (Week 12-Baseline), statistically controlling for Baseline levels.

    Baseline and End-of-Treatment (Treatment Week 12)

  • Clinical Global Impression-Improvement (CGI-I) at End-of-Treatment

    The Clinical Global Impression-Improvement (CGI-S) is rated by the patient's primary clinician on a scale of 1-7, with higher scores indicating greater impairment. Change will be measured as (Week 12-Baseline), statistically controlling for Baseline levels.

    End-of-Treatment (single administration at Treatment Week 12)

  • Peer Coach Fidelity Across Treatment Sessions

    Fidelity will be observer-rated by reliable coders to generate a fidelity ratio (0-100%), defined as the proportion of pre-defined critical session components administered to "acceptable" criterion; higher ratios indicate greater fidelity. The total fidelity score for each patient is calculated as the average of completed Baseline, Midpoint, and End-of-Treatment sessions.

    Calculated at End-of-Treatment (Treatment Week 12)

  • Coaching Session Completion

    Completion will be reported by project staff and used to generate a completion ratio (0-100%), defined as the proportion of sessions attended; higher ratios indicate greater completion.

    Calculated at End-of-Treatment (Treatment Week 12)

  • Treatment Session Completion

    Completion will be reported by clinicians to generate a completion ratio (0-100%), defined as the proportion of sessions attended; higher ratios indicate greater completion. Participants in the self-guided resource condition will self-report completion across 12 weeks of tasks using a log submitted at end of treatment.

    Calculated at End-of-Treatment (Treatment Week 12)

  • Peer Coaching Satisfaction

    Patient-reported peer coaching satisfaction scores are averaged across a study-specific feedback survey and range from 1-5; higher scores indicate greater satisfaction.

    End-of-Treatment (single administration at Treatment Week 12)

  • Treatment Satisfaction

    Patient-reported treatment satisfaction scores are averaged across a study-specific feedback survey and range from 1-5; higher scores indicate greater satisfaction.

    End-of-Treatment (single administration at Treatment Week 12)

  • Drop Out Status

    "Drop out" is defined as discontinuation of treatment after consenting to treatment and attending at least one session, including active discontinuation and patients lost to follow-up. Variable is dummy coded where 0=patient did not drop out, 1=patient dropped out.

    Calculated at End-of-Treatment (Treatment Week 12)

  • Homework Completion

    Completion will be reported by clinicians to generate a completion ratio (0-100%), defined as the proportion of assigned session homework completed; higher ratios indicate greater completion. Participants in the self-guided resource condition will self-report completion across 12 weeks of tasks using a log submitted at end of treatment.

    Calculated at End-of-Treatment (Treatment Week 12)

Secondary Outcomes (10)

  • Change from Baseline on EES-2 Total Score at End-of-Treatment Survey

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on DERS Total Score at End-of-Treatment Survey

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on CBCL Total Problem Behaviors at End-of-Treatment Survey

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on CDI-Words and Gestures at End-of-Treatment Survey

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change from Baseline on CSHQ at End-of-Treatment Survey

    Baseline and End-of-Treatment (Treatment Week 12)

  • +5 more secondary outcomes

Other Outcomes (4)

  • Change in Daily Reports of Health Behaviors between Baseline and End-of-Treatment

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change in Daily Reports of Caregiver Sleep between Baseline and End-of-Treatment

    Baseline and End-of-Treatment (Treatment Week 12)

  • Change in Momentary Self-Efficacy between Baseline and End-of-Treatment

    Baseline and End-of-Treatment (Treatment Week 12)

  • +1 more other outcomes

Study Arms (9)

No Algorithm + Participation Enhancement Intervention

EXPERIMENTAL

Participants are randomly assigned to support program without use of a personalized health algorithm AND receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided ResourcesBehavioral: Participation Enhancement Intervention (PEI)

No Algorithm + No Participation Enhancement Intervention

EXPERIMENTAL

Participants are randomly assigned to support program without use of a personalized health algorithm AND do not receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided Resources

Algorithm 1 + Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 1 AND receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided ResourcesBehavioral: Participation Enhancement Intervention (PEI)

Algorithm 1 + No Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 1 AND do not receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided Resources

Algorithm 2 + Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 2 AND receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided ResourcesBehavioral: Participation Enhancement Intervention (PEI)

Algorithm 2 + No Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 2 AND do not receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided Resources

Algorithm 3 + Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 3 AND receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided ResourcesBehavioral: Participation Enhancement Intervention (PEI)

Algorithm 3 + No Participation Enhancement Intervention

EXPERIMENTAL

Participants are assigned to support programs using Algorithm 3 AND do not receive the participation enhancement intervention.

Behavioral: Acceptance and Commitment Therapy (ACT)Behavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), IndividualBehavioral: Culturally Informed Cognitive Behavioral Therapy (CICBT), GroupBehavioral: Dialectical Behavioral Therapy (DBT)Behavioral: Durand Sleep InterventionBehavioral: RUBI InterventionBehavioral: Naturalistic Communication Intervention (NCI)Behavioral: Self-Guided Resources

Waitlist Control

NO INTERVENTION

Participants are enrolled in the waitlist control condition, which includes identical data collection procedures to participants enrolled in support programs. Waitlist controls are offered the opportunity to enroll in future cycles.

Interventions

ACT will be deployed weekly for 12 weeks on an individual basis via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed psychologist.

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

CICBT-Individual will be deployed weekly for 12 weeks on an individual basis via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed psychologist.

Also known as: Trauma Focused Cognitive Behavioral Therapy
Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

CICBT-Group will be deployed weekly for 12 weeks in small groups via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed psychologist.

Also known as: Trauma Focused Cognitive Behavioral Therapy
Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

DBT will be deployed weekly for 12 weeks in small groups via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed psychologist.

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

Durand Sleep Intervention will be deployed weekly for 12 weeks, alternating between individual and group formats, via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed psychologist or behavioral analyst.

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

RUBI will be deployed weekly for 12 weeks in small groups via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed behavioral analyst.

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

NCI will be deployed weekly for 12 weeks on an individual basis via secure Zoom. Treatments will be deployed by supervised graduate student clinicians under the supervision of a licensed behavioral analyst.

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

Participants will be provided a self-guided resource notebook that includes 12 weeks of content that parallels the topics examined in other support programs (child behavior management, child sleep, child communication, caregiver mental health, caregiver health behaviors, community connection)

Algorithm 1 + No Participation Enhancement InterventionAlgorithm 1 + Participation Enhancement InterventionAlgorithm 2 + No Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + No Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + No Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

A standardized PEI intervention will be conducted by trained peer coaches who are also rare disorder caregivers. Each peer coach will work with their clients to complete a "Change Plan Worksheet" and will complete two booster sessions at midpoint and end of treatment. Coaches will also meet with their clients after follow-up data are complete to support connection back to community resources and patient-specific rare disorder communities.

Algorithm 1 + Participation Enhancement InterventionAlgorithm 2 + Participation Enhancement InterventionAlgorithm 3 + Participation Enhancement InterventionNo Algorithm + Participation Enhancement Intervention

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Caregiver and legal guardian of child age 2-35 with neurogenetic condition
  • Child's syndrome must (1) have an established genetic cause, (2) affect the brain, resulting in moderate to severe intellectual disability in the majority of patients
  • Reside in US
  • Fluent in English (spoken and written)
  • Seeking support for caregiver mental health/well-being and/or caregiving needs

You may not qualify if:

  • Serious mental illness or active addiction that would be inadequately addressed through dosage of treatments
  • Actively in treatment that would be redundant with those offered in the protocol
  • Child's syndrome is not commonly associated with (1) life expectancy less than 35 years or (2) deterioration of previously gained skills.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Purdue University

West Lafayette, Indiana, 47907, United States

RECRUITING

Related Publications (3)

  • Kelleher BL. CASCADE: a community-engaged action model for generating rapid, patient-engaged decisions in clinical research. BMC Med Res Methodol. 2025 Jul 1;25(1):168. doi: 10.1186/s12874-025-02565-7.

  • Kelleher B, Emerson K, Graham LN, Vozka V, Wheeler A, Fadel W, Foti D, Metzger I, Rispoli M, Machalicek W, McLay L, Lane S, Neo WS, Carter A, Brown L, Brown J, McIntyre LL, Salwitz E, Dietz G, Naughton R, Peek K, Hollins N, Woodford E. Optimizing a Personalized Health Approach for Virtually Treating High-Risk Caregivers of Children With Neurogenetic Conditions (Project WellCAST): Protocol for a Randomized Controlled Trial. JMIR Res Protoc. 2025 Jun 25;14:e64360. doi: 10.2196/64360.

  • Kelleher BL. CASCADE: A Community-Engaged Action Model for Generating Rapid, Patient-Engaged Decisions in Clinical Research. Res Sq [Preprint]. 2024 Aug 27:rs.3.rs-4790564. doi: 10.21203/rs.3.rs-4790564/v1.

MeSH Terms

Interventions

Acceptance and Commitment TherapyPopulation Groups

Intervention Hierarchy (Ancestors)

Cognitive Behavioral TherapyBehavior TherapyPsychotherapyBehavioral Disciplines and ActivitiesDemographyPopulation Characteristics

Study Officials

  • Bridgette Kelleher, PhD

    Purdue University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Bridgette L Kelleher, PhD

CONTACT

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
CARE PROVIDER, OUTCOMES ASSESSOR
Masking Details
Care providers will be masked to the status of peer coaching and algorithm assignment. Peer coaches will be masked to the status of algorithm assignment. Data managers and statisticians evaluating study outcomes will be provided de-identified data and will not be involved in treatment-related study operations.
Purpose
TREATMENT
Intervention Model
FACTORIAL
Model Details: For Waves I-II, caregivers are randomly assigned to either (1) route to a treatment using their personalized health data ("Algorithm 1") OR route to treatment randomly ("No Algorithm"); for Waves III and IV, caregivers are randomly assigned to Algorithm 1 or an optimized Algorithm 2 (Wave III) or Algorithm 2 or an optimized and final Algorithm 3 (Wave IV). Across Waves I-IV, caregivers are randomly assigned to receive peer coaching ("Peer Coaching") or to receive check-ins by a staff member unfamiliar with the peer coaching protocol ("No Peer Coaching"). Across Waves I-III, a subset of caregivers are assigned to a waitlist control that does not include any treatment or peer coaching.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

July 17, 2023

First Posted

August 21, 2023

Study Start

June 28, 2023

Primary Completion

September 8, 2025

Study Completion

December 29, 2025

Last Updated

August 21, 2023

Record last verified: 2023-08

Data Sharing

IPD Sharing
Will share

IPD will be shared with other researchers using best-practices in open science.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
Time Frame
Primary outcome IPD will be shared no later than 12 months after study completion.
Access Criteria
Data access may be requested for eligible studies and qualified researchers for the purpose of addressing specific scientific objectives. A mutually signed Data Use Agreement (non-negotiable contract for data use and sharing) must be in place before accessing requested information. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with only information necessary to address the research objectives under the terms of the data sharing agreement.

Locations